Ignite Creation Date:
2025-12-26 @ 1:17 PM
Ignite Modification Date:
2026-01-13 @ 7:38 AM
Study NCT ID:
NCT02310906
Status:
COMPLETED
Last Update Posted:
2020-10-19
First Post:
2014-12-03
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Phase I/II Study of SRP-4053 in DMD Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@sarepta.com', 'phone': '1-800-690-2003', 'title': 'Medical Director', 'organization': 'Sarepta Therapeutics, Inc.'}, 'certainAgreement': {'otherDetails': 'The most restrictive relevant agreement provides that the PI can only publish the study results with the approval of Sponsor.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From start of study drug administration up to 189 weeks', 'eventGroups': [{'id': 'EG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen IV infusions, once weekly for up to 12 weeks in Part 1.', 'otherNumAtRisk': 4, 'deathsNumAtRisk': 4, 'otherNumAffected': 4, 'seriousNumAtRisk': 4, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG001', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 5, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 5, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 3, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG004', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.', 'otherNumAtRisk': 8, 'deathsNumAtRisk': 8, 'otherNumAffected': 6, 'seriousNumAtRisk': 8, 'deathsNumAffected': 0, 'seriousNumAffected': 0}, {'id': 'EG005', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 mg/kg once weekly, for up to 168 weeks in Part 2. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.', 'otherNumAtRisk': 25, 'deathsNumAtRisk': 25, 'otherNumAffected': 25, 'seriousNumAtRisk': 25, 'deathsNumAffected': 0, 'seriousNumAffected': 4}, {'id': 'EG006', 'title': 'Part 2b: Untreated Group (Natural History of Non-exon 53)', 'description': 'Untreated participants intended to evaluate the natural history of the disease with DMD and various genetic mutations (not amenable to exon 53 skipping) were included in this group and did not received any treatment. Participants underwent the same study assessments as treated participants in other reporting groups (except for PK sampling and muscle biopsies), but at a reduced schedule through Week 144. Thus, the untreated participants were not considered as control group.', 'otherNumAtRisk': 14, 'deathsNumAtRisk': 14, 'otherNumAffected': 10, 'seriousNumAtRisk': 14, 'deathsNumAffected': 0, 'seriousNumAffected': 2}], 'otherEvents': [{'term': 'Rhinitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 2}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 16}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 13}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 8}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 5}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 3}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Viral upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 3}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Conjunctivitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Lower respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Oral herpes', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Otitis media', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Bronchitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Device related infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Hordeolum', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Bronchiolitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Scarlet fever', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Cystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 15}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 14}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 9}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 1}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 2}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 9}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 5}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 4}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Dyspepsia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 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'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Skin papilloma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 3}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Haemangioma of skin', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Proteinuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Seasonal allergy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 2}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Immune system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Haematoma', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Flushing', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Hyperbilirubinaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}], 'seriousEvents': [{'term': 'Haematemesis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Tooth development disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Convulsion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Tonsillar hypertrophy', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 1}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}, {'term': 'Rhabdomyolysis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 4, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG004', 'numAtRisk': 8, 'numAffected': 0}, {'groupId': 'EG005', 'numAtRisk': 25, 'numAffected': 0}, {'groupId': 'EG006', 'numAtRisk': 14, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'NON_SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (17.1)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Discontinuation', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}, {'value': '8', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen IV infusions, once weekly for up to 12 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG004', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'title': 'Participants with TEAE', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '5', 'groupId': 'OG001'}, {'value': '5', 'groupId': 'OG002'}, {'value': '3', 'groupId': 'OG003'}, {'value': '6', 'groupId': 'OG004'}]}]}, {'title': 'Participants with Serious TEAE', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}, {'title': 'Participants with TEAEs leading to discontinuation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 12', 'description': 'Adverse event (AE) was any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. A Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. TEAEs were defined as AEs that were reported or worsened on or after the start of study drug dosing through 12 weeks. TEAEs included both Serious TEAEs and non-serious TEAEs.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Part 1 safety set included all randomized participants who received at least one dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part 1: Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}, {'value': '8', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen IV infusions, once weekly for up to 12 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG004', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'title': 'Hepatic Chemistry', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}, {'title': 'Renal Chemistry', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}, {'title': 'Hematology', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}, {'title': 'Coagulation', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}, {'title': 'Urinalysis', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 12', 'description': 'Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of participants with at least one potentially clinically significant abnormal finding were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potentially clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Part 1 safety set included all randomized participants who received at least one dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}, {'value': '8', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen IV infusions, once weekly for up to 12 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG004', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}, {'value': '3', 'groupId': 'OG001'}, {'value': '1', 'groupId': 'OG002'}, {'value': '1', 'groupId': 'OG003'}, {'value': '4', 'groupId': 'OG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 12', 'description': 'Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potential clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Part 1 safety set included all randomized participants who received at least one dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Physical Examinations', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}, {'value': '8', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen IV infusions, once weekly for up to 12 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG004', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '2', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '3', 'groupId': 'OG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 12', 'description': "Physical examinations were performed by the Investigator, or qualified study staff. A full physical examination included a review of general appearance, head, eyes, ears, nose and throat, heart, lungs, abdomen, extremities, skin, lymph nodes, musculoskeletal, and neurological systems. Number of participants with potentially clinically significant abnormalities in physical examinations were reported. Potentially clinically significant abnormalities in physical examinations were based on Investigator's discretion.", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Part 1 safety set included all randomized participants who received at least one dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}, {'value': '8', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen IV infusions, once weekly for up to 12 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG004', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '0', 'groupId': 'OG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 12', 'description': 'Twelve-lead ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECG reported as TEAEs presented here. The Investigator determined whether abnormal assessment results were potentially clinically significant or not.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Part 1 safety set included all randomized participants who received at least one dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Echocardiograms (ECHO)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}, {'value': '8', 'groupId': 'OG004'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen IV infusions, once weekly for up to 12 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG004', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}, {'value': '0', 'groupId': 'OG002'}, {'value': '0', 'groupId': 'OG003'}, {'value': '3', 'groupId': 'OG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline up to Week 12', 'description': 'Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECHO were reported.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Part 1 safety set included all randomized participants who received at least one dose of study drug.'}, {'type': 'PRIMARY', 'title': 'Part 2a: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) at Week 144 in Total Golodirsen Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 mg/kg once weekly, for up to 168 weeks in Part 2. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '-99.0', 'spread': '123.75', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': '6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 144 in total golodirsen group was reported.', 'unitOfMeasure': 'meters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy set consisted of all randomized participants who had at least one post baseline functional assessment. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure.'}, {'type': 'PRIMARY', 'title': 'Part 2b: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) at Week 144 in Untreated Group (Non-exon 53 Amenable Participants)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '6', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2b: Untreated Group (Natural History of Non-exon 53)', 'description': 'Untreated participants intended to evaluate the natural history of the disease with DMD and various genetic mutations (not amenable to exon 53 skipping) were included in this group and did not received any treatment. Participants underwent the same study assessments as treated participants in other reporting groups (except for PK sampling and muscle biopsies), but at a reduced schedule through Week 144. Thus, the untreated participants were not considered as control group.'}], 'classes': [{'categories': [{'measurements': [{'value': '-160.8', 'spread': '162.41', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline and Week 144', 'description': '6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 144 in untreated group (non-exon 53 amenable participants) was calculated.', 'unitOfMeasure': 'Meters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy set consisted of all randomized participants who had at least one post baseline functional assessment. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure.'}, {'type': 'PRIMARY', 'title': 'Part 2a: Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 48 in Total Golodirsen Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 mg/kg once weekly, for up to 168 weeks in Part 2. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.924', 'spread': '1.0129', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 48', 'description': 'Change from baseline in dystrophin protein levels (in muscle biopsy samples) was determined by Western blot in total golodirsen group.', 'unitOfMeasure': 'Percent normal dystrophin protein level', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Muscle biopsy set included all participants who received at least one dose of study drug and who had data from both baseline (pre-treatment) and Part 2 Week 48 (on-treatment) muscle biopsy samples. Data was not planned to be collected and analyzed for untreated group.'}, {'type': 'SECONDARY', 'title': 'Part 1: Maximum Plasma Concentration (Cmax) of Golodirsen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '7840', 'spread': '45.7', 'groupId': 'OG000'}, {'value': '17000', 'spread': '40.9', 'groupId': 'OG001'}, {'value': '39700', 'spread': '71.8', 'groupId': 'OG002'}, {'value': '53300', 'spread': '37.9', 'groupId': 'OG003'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Maximum Concentration (Cmax) of golodirsen in plasma was evaluated.', 'unitOfMeasure': 'nanogram per milliliter (ng/mL)', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'Pharmacokinetic (PK) set consisted of all randomized participants from Part 1 who received the planned full dose of study drug and for whom there were adequate PK samples from which to estimate PK parameters. Data were not planned to be collected and analyzed for the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Part 1: Time to Reach Maximum Plasma Concentration (Tmax) of Golodirsen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '8', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.11', 'groupId': 'OG000', 'lowerLimit': '0.77', 'upperLimit': '1.67'}, {'value': '1.09', 'groupId': 'OG001', 'lowerLimit': '0.12', 'upperLimit': '1.62'}, {'value': '1.12', 'groupId': 'OG002', 'lowerLimit': '0.80', 'upperLimit': '1.58'}, {'value': '1.12', 'groupId': 'OG003', 'lowerLimit': '0.68', 'upperLimit': '1.50'}]}]}], 'paramType': 'MEDIAN', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Time to reach maximum plasma concentration (Tmax) of golodirsen was evaluated.', 'unitOfMeasure': 'hour', 'dispersionType': 'Full Range', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set consisted of all randomized participants from Part 1 who received the planned full dose of study drug and for whom there were adequate PK samples from which to estimate PK parameters. Data were not planned to be collected and analyzed for the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Part 1: Area Under the Concentration-Time Curve From Time Zero Extrapolated to the Infinity (AUCinf) of Golodirsen in Plasma', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '11800', 'spread': '35.5', 'groupId': 'OG000'}, {'value': '26400', 'spread': '42.7', 'groupId': 'OG001'}, {'value': '62300', 'spread': '52.6', 'groupId': 'OG002'}, {'value': '90800', 'spread': '33.4', 'groupId': 'OG003'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Area under the concentration-time curve from time zero extrapolated to the infinity was evaluated.', 'unitOfMeasure': 'hr*ng/mL', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set: all randomized participants from Part 1 who received the planned full dose of study drug and for whom there were adequate PK samples from which to estimate PK parameters. "Overall number of participants analyzed"= number of participants evaluable for this outcome. Data were not planned to be collected and analyzed for the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Part 1: Apparent Volume of Distribution at Steady State (Vss) of Golodirsen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.670', 'spread': '38.6', 'groupId': 'OG000'}, {'value': '0.767', 'spread': '43.4', 'groupId': 'OG001'}, {'value': '0.576', 'spread': '84.5', 'groupId': 'OG002'}, {'value': '0.668', 'spread': '32.3', 'groupId': 'OG003'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Volume of distribution was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Apparent volume of distribution at steady state of golodirsen was evaluated.', 'unitOfMeasure': 'Liter per kilogram (L/kg)', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set: all randomized participants from Part 1 who received planned full dose of study drug, for whom there were adequate PK samples from which to estimate PK parameters. "Overall number of participants analyzed"= number of participants evaluable for this outcome. Data were not planned to be collected and analyzed for the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Part 1: Elimination Half-life (T1/2) of Golodirsen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.36', 'spread': '0.581', 'groupId': 'OG000'}, {'value': '3.63', 'spread': '2.04', 'groupId': 'OG001'}, {'value': '3.27', 'spread': '0.897', 'groupId': 'OG002'}, {'value': '3.42', 'spread': '0.628', 'groupId': 'OG003'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'T1/2 is the time measured for the plasma concentration of drug to decrease by one half. T1/2 of golodirsen was evaluated.', 'unitOfMeasure': 'hour', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set: all randomized participants from Part 1 who received planned full dose of study drug, for whom there were adequate PK samples from which to estimate PK parameters. "Overall number of participants analyzed"= number of participants evaluable for this outcome. Data were not planned to be collected and analyzed for the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Part 1: Total Clearance (CL) of Golodirsen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.381', 'spread': '36.0', 'groupId': 'OG000'}, {'value': '0.405', 'spread': '46.2', 'groupId': 'OG001'}, {'value': '0.338', 'spread': '52.2', 'groupId': 'OG002'}, {'value': '0.346', 'spread': '34.3', 'groupId': 'OG003'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.', 'unitOfMeasure': 'Liters per hour per kilogram (L/h/kg)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set: all randomized participants from Part 1 who received planned full dose of study drug, for whom there were adequate PK samples from which to estimate PK parameters. "Overall number of participants analyzed"= number of participants evaluable for this outcome. Data were not planned to be collected and analyzed for the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Part 1: Mean Residence Time (MRT) of Golodirsen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '6', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.79', 'spread': '0.325', 'groupId': 'OG000'}, {'value': '1.92', 'spread': '0.338', 'groupId': 'OG001'}, {'value': '1.77', 'spread': '0.495', 'groupId': 'OG002'}, {'value': '1.95', 'spread': '0.312', 'groupId': 'OG003'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'MRT= AUMCinf/AUCinf, where AUMCinf is the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method. Mean residence time of golodirsen was evaluated.', 'unitOfMeasure': 'hour', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set: all randomized participants from Part 1 who received planned full dose of study drug, for whom there were adequate PK samples from which to estimate PK parameters. "Overall number of participants analyzed"= number of participants evaluable for this outcome. Data were not planned to be collected and analyzed for the placebo arm'}, {'type': 'SECONDARY', 'title': 'Part 1: Renal Clearance (CLR) of Golodirsen', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}, {'value': '8', 'groupId': 'OG001'}, {'value': '7', 'groupId': 'OG002'}, {'value': '8', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 1: Golodirsen (4 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at dose levels of 4 mg/kg, once weekly for 2 weeks in Part 1.'}, {'id': 'OG001', 'title': 'Part 1: Golodirsen (10 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose levels of 10 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 4) in Part 1.'}, {'id': 'OG002', 'title': 'Part 1: Golodirsen (20 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 20 mg/kg, once weekly for the next 2 weeks (i.e., up to Week 6) in Part 1.'}, {'id': 'OG003', 'title': 'Part 1: Golodirsen (30 mg/kg)', 'description': 'Participants received golodirsen IV infusions, at a dose level of 30 mg/kg, once weekly from Week 7 to Week 12 in Part 1.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.345', 'spread': '31.9', 'groupId': 'OG000'}, {'value': '0.370', 'spread': '50.6', 'groupId': 'OG001'}, {'value': '0.355', 'spread': '42.1', 'groupId': 'OG002'}, {'value': '0.374', 'spread': '26.8', 'groupId': 'OG003'}]}]}], 'paramType': 'GEOMETRIC_MEAN', 'timeFrame': '0 to 1440 min after initiation of dosing on Day 1', 'description': 'Renal clearance was calculated using the partial AUC0-24 from the non-compartmental analysis in plasma and AE0-24. AUC0-24 was defined as area under the plasma concentration-time curve, from time 0 to 24 hours after completion of dosing. AE0-24 was defined as total cumulative amount excreted from 0 to 24 hours. Summarized data of all urine collection intervals are reported.', 'unitOfMeasure': 'L/h/kg', 'dispersionType': 'Geometric Coefficient of Variation', 'reportingStatus': 'POSTED', 'populationDescription': 'PK set: all randomized participants from Part 1 who received planned full dose of study drug, for whom there were adequate PK samples from which to estimate PK parameters. "Overall number of participants analyzed"= number of participants evaluable for this outcome. Data were not planned to be collected and analyzed for the placebo arm.'}, {'type': 'SECONDARY', 'title': 'Part 2a: Percent Change From Baseline in Forced Vital Capacity Predicted (FVC%p) at Week144 in Total Golodirsen Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '23', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 mg/kg once weekly, for up to 168 weeks in Part 2. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '-8.382', 'spread': '29.4566', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'FVC was the total amount of air exhaled during the forced expiratory volume test that was measured during spirometry and the most important measurement of lung function. This test required participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which was used to dilate participant bronchial (breathing) tubes. Percent of predicted FVC = (observed value)/ (predicted value) \\* 100%.', 'unitOfMeasure': 'Percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy set consisted of all randomized participants who had at least 1 post-baseline functional assessment. Here, "overall number of participants analyzed" = number of participants evaluable for this outcome.'}, {'type': 'SECONDARY', 'title': 'Part 2b: Percent Change From Baseline in Forced Vital Capacity Predicted (FVC%p) at Week144 in Untreated Group (Non-exon 53 Amenable Participants)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '5', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2b: Untreated Group (Natural History of Non-exon 53)', 'description': 'Untreated participants intended to evaluate the natural history of the disease with DMD and various genetic mutations (not amenable to exon 53 skipping) were included in this group and did not received any treatment. Participants underwent the same study assessments as treated participants in other reporting groups (except for PK sampling and muscle biopsies), but at a reduced schedule through Week 144.Thus, the untreated participants were not considered as control group.'}], 'classes': [{'categories': [{'measurements': [{'value': '-6.739', 'spread': '17.5278', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'FVC was the total amount of air exhaled during the forced expiratory volume test that was measured during spirometry and is the most important measurement of lung function. This test requires participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which was used to dilate participant bronchial (breathing) tubes. Percent of predicted FVC = (observed value)/ (predicted value) \\* 100%.', 'unitOfMeasure': 'Percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Efficacy set consisted of all randomized participants who had at least 1 post-baseline functional assessment. Here, "overall number of participants analyzed" = number of participants evaluable for this outcome.'}, {'type': 'SECONDARY', 'title': 'Part 2a: Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 in Total Golodirsen Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 mg/kg once weekly, for up to 168 weeks in Part 2. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.023', 'spread': '0.0243', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 48', 'description': 'Change from baseline in dystrophin Intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry in total golodirsen group.', 'unitOfMeasure': 'Percent dystrophin positive fibers', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Muscle biopsy set included all participants who received at least 1 dose of study drug and who had data from both baseline (pre-treatment) and Part 2 Week 48 (on-treatment) muscle biopsy samples. Data were not planned to be collected and analyzed for the untreated group.'}, {'type': 'SECONDARY', 'title': 'Part 2a: Percent Change From Baseline in Exon 53 Skipping Determined by Reverse Transcription Polymerase Chain Reaction (PCR) at Week 48 in Total Golodirsen Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 mg/kg once weekly, for up to 168 weeks in Part 2. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '16.363', 'spread': '10.6223', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 48', 'description': 'Percent change from baseline in Exon 53 skipping (in muscle biopsy samples) was determined by reverse transcription polymerase chain reaction in total golodirsen group.', 'unitOfMeasure': 'Percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Muscle biopsy set included all participants who received at least 1 dose of study drug and who had data from both baseline (pre-treatment) and Part 2 Week 48 (on-treatment) muscle biopsy samples. Data were not planned to be collected and analyzed for the untreated group.'}, {'type': 'SECONDARY', 'title': 'Part 2a: Percent Change From Baseline in Dystrophin Positive Fibers Determined by Immunohistochemistry (IHC) at Week 48 in Total Golodirsen Group', 'denoms': [{'units': 'Participants', 'counts': [{'value': '25', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 mg/kg once weekly, for up to 168 weeks in Part 2. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}], 'classes': [{'categories': [{'measurements': [{'value': '12.508', 'spread': '14.6012', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 48', 'description': 'Percent change from baseline in dystrophin positive fibers (in muscle biopsy samples) were determined by Immunohistochemistry at Week 48 in total golodirsen group.', 'unitOfMeasure': 'Percent change', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Muscle biopsy set included all participants who received at least 1 dose of study drug and who had data from both baseline (pre-treatment) and Part 2 Week 48 (on-treatment) muscle biopsy samples. Data were not planned to be collected and analyzed for the untreated group.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen intravenous (IV) infusions, once weekly up to 12 weeks in Part 1.'}, {'id': 'FG001', 'title': 'Part 1: Golodirsen', 'description': 'Participants received golodirsen IV infusions, at four dose levels of 4 milligrams per kilograms (mg/kg) once weekly for 2 weeks, followed by 10 mg/kg once weekly for the next 2 weeks (i.e., up to Week 4), followed by 20 mg/kg once weekly for the next 2 weeks (i.e., up to Week 6), followed by 30 mg/kg once weekly from Week 7 to Week 12 in Part 1.'}, {'id': 'FG002', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 milligram (mg)/kilogram (kg) once weekly, for up to 168 weeks. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}, {'id': 'FG003', 'title': 'Part 2b: Untreated Group (Natural History of Non-exon 53)', 'description': 'Untreated participants intended to evaluate the natural history of the disease with DMD and various genetic mutations (not amenable to exon 53 skipping) were included in this group and did not received any treatment. Participants underwent the same study assessments as treated participants in other reporting groups (except for pharmacokinetic \\[PK\\] sampling and muscle biopsies), but at a reduced schedule through Week 144. Thus, the untreated participants were not considered as control group.'}], 'periods': [{'title': 'Part 1: Double Blind Phase (12 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Received 4 mg/kg', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Received 10 mg/kg', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Received 20 mg/kg', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Received 30 mg/kg', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '4'}, {'groupId': 'FG001', 'numSubjects': '8'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}, {'title': 'Part 2: Open Label Phase (168 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'comment': '4 participants from Part 1(Placebo) + 8 participants from Part 1(Golodirsen) +13 new participants', 'groupId': 'FG002', 'numSubjects': '25'}, {'groupId': 'FG003', 'numSubjects': '14'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '23'}, {'groupId': 'FG003', 'numSubjects': '6'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '2'}, {'groupId': 'FG003', 'numSubjects': '8'}]}], 'dropWithdraws': [{'type': 'Enrollment in other therapeutic study', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '2'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '2'}, {'groupId': 'FG003', 'numSubjects': '4'}]}, {'type': 'Personal reasons', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '1'}]}, {'type': 'Lost to Follow-up', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '1'}]}]}], 'recruitmentDetails': 'The study was conducted at 5 sites in the France, Italy, United Kingdom and United States from 13 January 2015 to 25 March 2019.', 'preAssignmentDetails': 'Study conducted in 2 parts: Part 1 and Part 2. When Part 1 was completed and cumulative safety data was reviewed by an independent Data Safety Monitoring Board (DSMB), Part 2 was conducted.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}, {'value': '51', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': 'Part 1: Placebo', 'description': 'Participants received placebo-matched to golodirsen intravenous (IV) infusions, once weekly up to 12 weeks in Part 1.'}, {'id': 'BG001', 'title': 'Part 1: Golodirsen', 'description': 'Participants received golodirsen IV infusions, at four dose levels of 4 milligrams per kilograms (mg/kg) once weekly for 2 weeks, followed by 10 mg/kg once weekly for the next 2 weeks (i.e., up to Week 4), followed by 20 mg/kg once weekly for the next 2 weeks (i.e., up to Week 6), followed by 30 mg/kg once weekly from Week 7 to Week 12 in Part 1.'}, {'id': 'BG002', 'title': 'Part 2a: Total Golodirsen Group', 'description': 'All participants from Part 1 (who previously received placebo or golodirsen) and including additional new participants received golodirsen 30 milligram (mg)/kilogram (kg) once weekly, for up to 168 weeks. Dosing was interrupted or halted when any specific predefined stopping criteria was met or if warranted at the discretion of the Sponsor or Investigator.'}, {'id': 'BG003', 'title': 'Part 2b: Untreated Group (Natural History of Non-exon 53)', 'description': 'Untreated participants intended to evaluate the natural history of the disease with DMD and various genetic mutations (not amenable to exon 53 skipping) were included in this group and did not received any treatment. Participants underwent the same study assessments as treated participants in other reporting groups (except for PK sampling and muscle biopsies), but at a reduced schedule through Week 144. Thus, the untreated participants were not considered as control group.'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'title': 'Part 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '12', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '7.0', 'spread': '0.82', 'groupId': 'BG000'}, {'value': '8.6', 'spread': '2.07', 'groupId': 'BG001'}, {'value': '8.1', 'spread': '1.88', 'groupId': 'BG004'}]}]}, {'title': 'Part 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}, {'value': '39', 'groupId': 'BG004'}]}], 'categories': [{'measurements': [{'value': '8.4', 'spread': '2.18', 'groupId': 'BG002'}, {'value': '8.5', 'spread': '1.91', 'groupId': 'BG003'}, {'value': '8.4', 'spread': '2.06', 'groupId': 'BG004'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'All participants who were previously randomized in Part 1 were moved to Part 2a (after completion of Part 1, n=12) along with 13 new participants. Hence, total number of participants in Part 2a were 25. In Part 2b, 14 untreated new participants were enrolled.'}, {'title': 'Sex: Female, Male', 'classes': [{'title': 'Part 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '12', 'groupId': 'BG004'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG004'}]}]}, {'title': 'Part 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}, {'value': '39', 'groupId': 'BG004'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '25', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}, {'value': '39', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'All participants who were previously randomized in Part 1 were moved to Part 2a (after completion of Part 1, n=12) along with 13 new participants. Hence, total number of participants in Part 2a were 25. In Part 2b, 14 untreated new participants were enrolled.'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'title': 'Part 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '12', 'groupId': 'BG004'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '2', 'groupId': 'BG004'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG004'}]}]}, {'title': 'Part 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}, {'value': '39', 'groupId': 'BG004'}]}], 'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '4', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '4', 'groupId': 'BG004'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '9', 'groupId': 'BG002'}, {'value': '9', 'groupId': 'BG003'}, {'value': '18', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '12', 'groupId': 'BG002'}, {'value': '5', 'groupId': 'BG003'}, {'value': '17', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'All participants who were previously randomized in Part 1 were moved to Part 2a (after completion of Part 1, n=12) along with 13 new participants. Hence, total number of participants in Part 2a were 25. In Part 2b, 14 untreated new participants were enrolled.'}, {'title': 'Race (NIH/OMB)', 'classes': [{'title': 'Part 1', 'denoms': [{'units': 'Participants', 'counts': [{'value': '4', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '12', 'groupId': 'BG004'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'White', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG004'}]}, {'title': 'More than one race', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG004'}]}]}, {'title': 'Part 2', 'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '25', 'groupId': 'BG002'}, {'value': '14', 'groupId': 'BG003'}, {'value': '39', 'groupId': 'BG004'}]}], 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Asian', 'measurements': [{'value': '0', 'groupId': 'BG002'}, {'value': '1', 'groupId': 'BG003'}, {'value': '1', 'groupId': 'BG004'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'White', 'measurements': [{'value': '23', 'groupId': 'BG002'}, {'value': '11', 'groupId': 'BG003'}, {'value': '34', 'groupId': 'BG004'}]}, {'title': 'More than one race', 'measurements': [{'value': '2', 'groupId': 'BG002'}, {'value': '2', 'groupId': 'BG003'}, {'value': '4', 'groupId': 'BG004'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'All participants who were previously randomized in Part 1 were moved to Part 2a (after completion of Part 1, n=12) along with 13 new participants. Hence, total number of participants in Part 2a were 25. In Part 2b, 14 untreated new participants were enrolled.'}], 'populationDescription': 'Safety set included all randomized participants from Part 1 and all Part 2 participants amenable to exon 53 skipping who received any amount of study drug, and all untreated participants who entered Part 2.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-11-08', 'size': 2535594, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-09-09T06:02', 'hasProtocol': True}, {'date': '2018-05-30', 'size': 4303787, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-09-09T06:04', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Masking Description: Part 1 is double-blind and randomized; Part 2 is open-label.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 39}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2015-01-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-10', 'completionDateStruct': {'date': '2019-03-25', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-10-14', 'studyFirstSubmitDate': '2014-12-03', 'resultsFirstSubmitDate': '2020-09-09', 'studyFirstSubmitQcDate': '2014-12-05', 'lastUpdatePostDateStruct': {'date': '2020-10-19', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-10-14', 'studyFirstPostDateStruct': {'date': '2014-12-08', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-10-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2019-03-25', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part 1: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Discontinuation', 'timeFrame': 'Baseline up to Week 12', 'description': 'Adverse event (AE) was any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. A Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. TEAEs were defined as AEs that were reported or worsened on or after the start of study drug dosing through 12 weeks. TEAEs included both Serious TEAEs and non-serious TEAEs.'}, {'measure': 'Part 1: Number of Participants With Potentially Clinically Significant (PCS) Laboratory Abnormalities Reported as TEAEs', 'timeFrame': 'Baseline up to Week 12', 'description': 'Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of participants with at least one potentially clinically significant abnormal finding were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potentially clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.'}, {'measure': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs', 'timeFrame': 'Baseline up to Week 12', 'description': 'Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were potentially clinically significant or not. Potential clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care.'}, {'measure': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Physical Examinations', 'timeFrame': 'Baseline up to Week 12', 'description': "Physical examinations were performed by the Investigator, or qualified study staff. A full physical examination included a review of general appearance, head, eyes, ears, nose and throat, heart, lungs, abdomen, extremities, skin, lymph nodes, musculoskeletal, and neurological systems. Number of participants with potentially clinically significant abnormalities in physical examinations were reported. Potentially clinically significant abnormalities in physical examinations were based on Investigator's discretion."}, {'measure': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Electrocardiogram (ECG) Reported as TEAEs', 'timeFrame': 'Baseline up to Week 12', 'description': 'Twelve-lead ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECG reported as TEAEs presented here. The Investigator determined whether abnormal assessment results were potentially clinically significant or not.'}, {'measure': 'Part 1: Number of Participants With Potentially Clinically Significant Abnormalities in Echocardiograms (ECHO)', 'timeFrame': 'Baseline up to Week 12', 'description': 'Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with potentially clinically significant abnormalities in ECHO were reported.'}, {'measure': 'Part 2a: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) at Week 144 in Total Golodirsen Group', 'timeFrame': 'Baseline and Week 144', 'description': '6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 144 in total golodirsen group was reported.'}, {'measure': 'Part 2b: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) at Week 144 in Untreated Group (Non-exon 53 Amenable Participants)', 'timeFrame': 'Baseline and Week 144', 'description': '6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course of 25 meters for 6 minutes (timed), and the distance walked (in meters) was recorded. Change from baseline in 6MWT distance at Week 144 in untreated group (non-exon 53 amenable participants) was calculated.'}, {'measure': 'Part 2a: Change From Baseline in Dystrophin Protein Levels Determined by Western Blot at Week 48 in Total Golodirsen Group', 'timeFrame': 'Baseline, Week 48', 'description': 'Change from baseline in dystrophin protein levels (in muscle biopsy samples) was determined by Western blot in total golodirsen group.'}], 'secondaryOutcomes': [{'measure': 'Part 1: Maximum Plasma Concentration (Cmax) of Golodirsen', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Maximum Concentration (Cmax) of golodirsen in plasma was evaluated.'}, {'measure': 'Part 1: Time to Reach Maximum Plasma Concentration (Tmax) of Golodirsen', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Time to reach maximum plasma concentration (Tmax) of golodirsen was evaluated.'}, {'measure': 'Part 1: Area Under the Concentration-Time Curve From Time Zero Extrapolated to the Infinity (AUCinf) of Golodirsen in Plasma', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Area under the concentration-time curve from time zero extrapolated to the infinity was evaluated.'}, {'measure': 'Part 1: Apparent Volume of Distribution at Steady State (Vss) of Golodirsen', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Volume of distribution was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of a drug. Apparent volume of distribution at steady state of golodirsen was evaluated.'}, {'measure': 'Part 1: Elimination Half-life (T1/2) of Golodirsen', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'T1/2 is the time measured for the plasma concentration of drug to decrease by one half. T1/2 of golodirsen was evaluated.'}, {'measure': 'Part 1: Total Clearance (CL) of Golodirsen', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.'}, {'measure': 'Part 1: Mean Residence Time (MRT) of Golodirsen', 'timeFrame': 'Pre-dose, 5 to 10 minutes, 1, 1.5, 2, 4, 6, 8, 12, 16 and 24 hours post-dose at Weeks 1 (for 4 mg/kg arm), 3 (for 10 mg/kg arm), 5 (for 20 mg/kg arm) and 7 (for 30 mg/kg arm)', 'description': 'MRT= AUMCinf/AUCinf, where AUMCinf is the area under the first moment curve from time 0 extrapolated to infinite time, calculated using the linear/log trapezoidal method. Mean residence time of golodirsen was evaluated.'}, {'measure': 'Part 1: Renal Clearance (CLR) of Golodirsen', 'timeFrame': '0 to 1440 min after initiation of dosing on Day 1', 'description': 'Renal clearance was calculated using the partial AUC0-24 from the non-compartmental analysis in plasma and AE0-24. AUC0-24 was defined as area under the plasma concentration-time curve, from time 0 to 24 hours after completion of dosing. AE0-24 was defined as total cumulative amount excreted from 0 to 24 hours. Summarized data of all urine collection intervals are reported.'}, {'measure': 'Part 2a: Percent Change From Baseline in Forced Vital Capacity Predicted (FVC%p) at Week144 in Total Golodirsen Group', 'timeFrame': 'Baseline, Week 144', 'description': 'FVC was the total amount of air exhaled during the forced expiratory volume test that was measured during spirometry and the most important measurement of lung function. This test required participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which was used to dilate participant bronchial (breathing) tubes. Percent of predicted FVC = (observed value)/ (predicted value) \\* 100%.'}, {'measure': 'Part 2b: Percent Change From Baseline in Forced Vital Capacity Predicted (FVC%p) at Week144 in Untreated Group (Non-exon 53 Amenable Participants)', 'timeFrame': 'Baseline, Week 144', 'description': 'FVC was the total amount of air exhaled during the forced expiratory volume test that was measured during spirometry and is the most important measurement of lung function. This test requires participant to breath into a tube connected to a machine that measures the amount of air that can be moved in and out of the lungs after taking an inhaled bronchodilator medicine which was used to dilate participant bronchial (breathing) tubes. Percent of predicted FVC = (observed value)/ (predicted value) \\* 100%.'}, {'measure': 'Part 2a: Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 in Total Golodirsen Group', 'timeFrame': 'Baseline, Week 48', 'description': 'Change from baseline in dystrophin Intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry in total golodirsen group.'}, {'measure': 'Part 2a: Percent Change From Baseline in Exon 53 Skipping Determined by Reverse Transcription Polymerase Chain Reaction (PCR) at Week 48 in Total Golodirsen Group', 'timeFrame': 'Baseline, Week 48', 'description': 'Percent change from baseline in Exon 53 skipping (in muscle biopsy samples) was determined by reverse transcription polymerase chain reaction in total golodirsen group.'}, {'measure': 'Part 2a: Percent Change From Baseline in Dystrophin Positive Fibers Determined by Immunohistochemistry (IHC) at Week 48 in Total Golodirsen Group', 'timeFrame': 'Baseline, Week 48', 'description': 'Percent change from baseline in dystrophin positive fibers (in muscle biopsy samples) were determined by Immunohistochemistry at Week 48 in total golodirsen group.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Duchenne muscular dystrophy', 'Exon Skipping', 'DMD', 'Exon 53', 'Ambulatory', 'Pediatric'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'references': [{'pmid': '32139505', 'type': 'BACKGROUND', 'citation': 'Frank DE, Schnell FJ, Akana C, El-Husayni SH, Desjardins CA, Morgan J, Charleston JS, Sardone V, Domingos J, Dickson G, Straub V, Guglieri M, Mercuri E, Servais L, Muntoni F; SKIP-NMD Study Group. Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy. Neurology. 2020 May 26;94(21):e2270-e2282. doi: 10.1212/WNL.0000000000009233. Epub 2020 Mar 5.'}, {'pmid': '34788571', 'type': 'DERIVED', 'citation': 'Servais L, Mercuri E, Straub V, Guglieri M, Seferian AM, Scoto M, Leone D, Koenig E, Khan N, Dugar A, Wang X, Han B, Wang D, Muntoni F; SKIP-NMD Study Group. Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial. Nucleic Acid Ther. 2022 Feb;32(1):29-39. doi: 10.1089/nat.2021.0043. Epub 2021 Nov 17.'}]}, 'descriptionModule': {'briefSummary': 'This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.', 'detailedDescription': 'Part 1: Randomized, placebo-controlled dose-titration to assess safety, tolerability and pharmacokinetics of 4 dose levels of SRP-4053 in genotypically-confirmed DMD patients with deletions amenable to exon 53 skipping.\n\nPart 2: Open-label evaluation of SRP-4053 in patients from Part 1, along with newly enrolled DMD patients with deletions amenable to exon 53 skipping and an untreated group of DMD patients with deletions not amenable to exon 53 skipping.\n\nSafety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients.\n\nClinical efficacy, including functional tests such as the six-minute walk test (6MWT), will be assessed at regularly scheduled study visits. Patients in the treated groups will undergo one baseline and one follow-up muscle biopsy. Patients in the untreated group will not undergo biopsies and will follow an abbreviated schedule of study assessments.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '15 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosed with DMD, genotypically confirmed.\n* Intact right and left biceps muscles or an alternative upper arm muscle group.\n* Stable pulmonary and cardiac function.\n* Minimum performance on 6MWT, North Star Ambulatory Assessment, and rise (Gowers) test as specified in the study protocol.\n* On a stable dose of corticosteroids for at least 6 months.\n\nExclusion Criteria:\n\n* Previous treatment with the experimental agents BMN-195 (SMT C1100) or PRO053.\n* Current or previous treatment with any other experimental treatments within 12 weeks prior to study entry.\n* Major surgery within the last 3 months.\n* Presence of other clinically significant illness.\n* Major change in physical therapy regime within the last 3 months.\n\nOther inclusion and exclusion criteria may apply.'}, 'identificationModule': {'nctId': 'NCT02310906', 'briefTitle': 'Phase I/II Study of SRP-4053 in DMD Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sarepta Therapeutics, Inc.'}, 'officialTitle': 'A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping', 'orgStudyIdInfo': {'id': '4053-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part 1: SRP-4053', 'description': 'Patients will receive SRP-4053 (golodirsen) intravenous (IV) infusions, weekly, at escalating dose levels as follows: Weeks 1-2, 4 mg/kg/week; Weeks 3-4, 10 mg/kg/week; Weeks 5-6, 20 mg/kg/week; Weeks 7-12, 30 mg/kg/week. Dosing will be interrupted or halted if specific predefined stopping criteria are met or if warranted at the discretion of the Sponsor or Investigator.', 'interventionNames': ['Drug: SRP-4053']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Part 1: Placebo', 'description': 'Patients will receive SRP-4053 placebo-matching IV infusions, weekly, for 12 weeks. Dosing will be interrupted or halted if specific predefined stopping criteria are met or if warranted at the discretion of the Sponsor or Investigator.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Part 2: SRP-4053', 'description': 'All eligible patients from Part 1, as well as new patients, will receive SRP-4053 (golodirsen) 30 mg/kg/week IV infusions, weekly, for up to 168 weeks.', 'interventionNames': ['Drug: SRP-4053']}, {'type': 'NO_INTERVENTION', 'label': 'Part 2: Untreated Group', 'description': 'Patients with DMD who have a genotypically confirmed deletion of exon(s) not amenable to treatment by exon 53 skipping, but who otherwise meet the same eligibility criteria as treated patients newly recruited to Part 2, will undergo the same study assessments as treated Patients (except for pharmacokinetic \\[PK\\] sampling and muscle biopsies), but at a reduced schedule through Week 144. The untreated patients are not considered as control group.'}], 'interventions': [{'name': 'Placebo', 'type': 'DRUG', 'description': 'SRP-4053 placebo-matching solution for IV infusion.', 'armGroupLabels': ['Part 1: Placebo']}, {'name': 'SRP-4053', 'type': 'DRUG', 'description': 'SRP-4053 (golodirsen) solution for IV infusion.', 'armGroupLabels': ['Part 1: SRP-4053', 'Part 2: SRP-4053']}]}, 'contactsLocationsModule': {'locations': [{'zip': '02116', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '75013', 'city': 'Paris', 'country': 'France', 'facility': 'Institute de Myologie', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '00168', 'city': 'Rome', 'country': 'Italy', 'facility': 'Policlinico Universitario A Gemelli', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'zip': 'WC1N 3JH', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital for Children NHS Foundation Trust', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'city': 'Newcastle', 'country': 'United Kingdom', 'facility': 'Newcastle University Hospital', 'geoPoint': {'lat': 54.21804, 'lon': -5.88979}}], 'overallOfficials': [{'name': 'Medical Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Sarepta Therapeutics, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sarepta Therapeutics, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Institut de Myologie, France', 'class': 'OTHER'}, {'name': 'Consultants for Research in Imaging and Spectroscopy', 'class': 'OTHER'}, {'name': 'Great Ormond Street Hospital for Children NHS Foundation Trust', 'class': 'OTHER'}, {'name': 'Catholic University of the Sacred Heart', 'class': 'OTHER'}, {'name': 'Royal Holloway University', 'class': 'OTHER'}, {'name': 'SYSNAV', 'class': 'INDUSTRY'}, {'name': 'University College, London', 'class': 'OTHER'}, {'name': 'University of Newcastle Upon-Tyne', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}