Ignite Creation Date:
2025-12-24 @ 3:06 PM
Ignite Modification Date:
2025-12-25 @ 1:27 PM
Study NCT ID:
NCT02090959
Status:
TERMINATED
Last Update Posted:
2020-08-11
First Post:
2014-03-17
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}, {'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'}], 'ancestors': [{'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C515878', 'term': 'ataluren'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@ptcbio.com', 'phone': '1-866-562-4620', 'title': 'Patient Advocacy', 'organization': 'PTC Therapeutics, Inc.'}, 'certainAgreement': {'otherDetails': 'The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}, 'limitationsAndCaveats': {'description': 'The study was terminated early per Sponsor decision due to commercial availability of ataluren.'}}, 'adverseEventsModule': {'timeFrame': 'Baseline (Day 1) up to 6 weeks post-treatment (Week 150)', 'description': 'AT population included all participants who received at least 1 dose of ataluren treatment in this extension study.', 'eventGroups': [{'id': 'EG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.', 'otherNumAtRisk': 218, 'deathsNumAtRisk': 218, 'otherNumAffected': 201, 'seriousNumAtRisk': 218, 'deathsNumAffected': 0, 'seriousNumAffected': 24}], 'otherEvents': [{'term': 'Ear infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 11}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 18}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 23}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 57}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 28}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 48}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 19}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Disease progression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 56}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 26}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Abdominal pain upper', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 18}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 17}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 16}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 37}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 14}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 27}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 26}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 26}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 42}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Haematuria', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 20}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}], 'seriousEvents': [{'term': 'Exposure to communicable disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Femur fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 5}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hip fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Lumbar vertebral fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Spinal compression fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Adenoiditis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Appendicitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Chronic sinusitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Varicella', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Colitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Colitis ulcerative', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Gastritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Intussusception', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Flank pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Joint contracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Tendinous contracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Acute respiratory distress syndrome', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hypoxia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 2}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pneumonia aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Pulmonary artery stenosis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Bradycardia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Transposition of the great vessels', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Ventricular septal defect', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Congenital, familial and genetic disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Cataract', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Eye disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Drug hypersensitivity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Immune system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hypocalcaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Nephrolithiasis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Renal and urinary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}, {'term': 'Hypotension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 218, 'numAffected': 1}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 20.1'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'title': 'Any AEs', 'categories': [{'measurements': [{'value': '202', 'groupId': 'OG000'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '24', 'groupId': 'OG000'}]}]}, {'title': 'Drug-Related AEs', 'categories': [{'measurements': [{'value': '44', 'groupId': 'OG000'}]}]}, {'title': 'AEs Leading to Withdrawal From Study', 'categories': [{'measurements': [{'value': '1', 'groupId': 'OG000'}]}]}, {'title': 'Mild AEs', 'categories': [{'measurements': [{'value': '87', 'groupId': 'OG000'}]}]}, {'title': 'Moderate AEs', 'categories': [{'measurements': [{'value': '71', 'groupId': 'OG000'}]}]}, {'title': 'Severe AEs', 'categories': [{'measurements': [{'value': '42', 'groupId': 'OG000'}]}]}, {'title': 'Life-threatening AEs', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline (Day 1) up to 6 weeks post-treatment (Week 150)', 'description': 'An adverse event (AE): any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of AEs: graded per Common Terminology Criteria for AEs (CTCAE), Version 3.0 as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). Drug-related AEs: AEs with possible, probable, unlikely relationship, or unrelated to study drug. Serious AEs (SAEs): death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: an AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all SAEs, regardless of causality is located in Reported AE section.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'AT population included all participants who received at least 1 dose of ataluren treatment in this extension study.'}, {'type': 'PRIMARY', 'title': 'Number of Participants With Abnormalities in Clinical Laboratory Parameters', 'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '29', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline (Day 1) up to 6 weeks post-treatment (Week 150)', 'description': 'Abnormalities in laboratory variables as pre-defined in protocol for safety-monitoring were: Hepatic (Serum alanine aminotransferase \\[ALT\\]: increase of greater than \\[\\>\\] 150 units/liter \\[U/L\\] with stable or decrease of creatinine kinese \\[CK\\]; Serum glutamyl amino transferase \\[GGT\\] \\[U/L\\]: Grade 2 \\[\\>2.5 - 5.0 \\* upper limit of normal {ULN}\\]), renal (Serum cystatin C miiligrams/liter \\[mg/L\\] \\>1.33 - 2.00 mg/L; Serum blood urea nitrogen \\[UREAN\\] \\[millimoles/liter {mmol/L}\\] greater than or equal to \\[≥\\]1.5 - 3.0 \\* ULN; Urine occult blood: 2+ \\[Small\\], 3+ \\[Moderate\\], 4+ \\[Large\\]), and electrolytes (Serum sodium: low \\[mmol/L\\], Grade 3-4 \\[less than {\\<}130 mmol/L\\]; serum potassium: high \\[mmol/L\\], Grade 3-4 \\[\\>6.0 mmol/L\\]; and Serum bicarbonate \\[mmol/L\\]: Grade 2 \\[\\<16 - 11 mmol/L\\]).', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'AT population included all participants who received at least 1 dose of ataluren treatment in this extension study.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in 6MWD at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '26', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-98.18', 'spread': '86.604', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'The 6MWD test was performed in a 30 meters long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.', 'unitOfMeasure': 'meters', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline 6MWD value at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Time to Stand From Supine Position at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '19', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '5.22', 'spread': '5.104', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Time to Walk/Run 10 Meters at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '27', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.29', 'spread': '1.991', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Time to Climb 4 Stairs at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '4.01', 'spread': '7.260', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Time to Descend 4 Stairs at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '22', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '2.45', 'spread': '5.853', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.', 'unitOfMeasure': 'seconds', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '40', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-7.95', 'spread': '5.611', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one\'s head, stand on one\'s heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for \'raise one\'s head\' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in PUL Total Score at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '44', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-4.0', 'spread': '7.49', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'The PUL was used to assess motor performance of the upper limb. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into shoulder level (4 items), elbow level (9 items), and distal level (8 items) dimensions. Scoring options per item may not be uniform and may vary from 0-1 and 0-6, according to the performance, with higher values corresponding to better performance. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for elbow level, and 24 for distal level. Total score was calculated by adding the 3 level scores, with a maximum global score of 74 (total score range = 0-74). Higher score = better outcome.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '3.51', 'spread': '14.253', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'FVC is a standard pulmonary function test. FVC was defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Percent predicted FVC (in %) = \\[(observed FVC)/(predicted FVC)\\]\\*100.', 'unitOfMeasure': 'percent predicted FVC', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Percent Predicted FEV1 as Measured by Spirometry at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '1.40', 'spread': '15.319', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'FEV1 is a standard pulmonary function test. FEV1 was defined as the volume of air that can forcibly be blown out in 1 second, after full inspiration in the upright position, measured in liters. Percent predicted FEV1 (in %) = \\[(observed FEV1)/(predicted FEV1)\\]\\*100.', 'unitOfMeasure': 'percent predicted FEV1', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in PEF as Measured by Spirometry at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '65', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.23', 'spread': '1.099', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'PEF was defined as the maximum airflow during a forced expiration beginning with the lungs fully inflated.', 'unitOfMeasure': 'L/sec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in PCF as Measured by Spirometry at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '57', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.53', 'spread': '1.281', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': "PCF measures an individual's maximum speed of expiration during cough.", 'unitOfMeasure': 'L/sec', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in PODCI Transfers/Basic Mobility Score at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '65', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-29.3', 'spread': '25.05', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domain was prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 144, as Assessed by a Standardized Survey Administered by Site Personnel', 'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'title': 'Physical Functioning: Walking', 'denoms': [{'units': 'Participants', 'counts': [{'value': '143', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '6', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '3', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '47', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '32', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '55', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Climbing Stairs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '141', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '6', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '4', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '37', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '29', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '65', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Upper Extremity Activity', 'denoms': [{'units': 'Participants', 'counts': [{'value': '119', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '5', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '6', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '75', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '21', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '12', 'groupId': 'OG000'}]}]}, {'title': 'Physical Functioning: Other', 'denoms': [{'units': 'Participants', 'counts': [{'value': '62', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '3', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '29', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '13', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '15', 'groupId': 'OG000'}]}]}, {'title': 'Emotional/Social Functioning', 'denoms': [{'units': 'Participants', 'counts': [{'value': '134', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '13', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '16', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '93', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '7', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '5', 'groupId': 'OG000'}]}]}, {'title': 'Cognition/Social Functioning', 'denoms': [{'units': 'Participants', 'counts': [{'value': '133', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '9', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '24', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '92', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '8', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '0', 'groupId': 'OG000'}]}]}, {'title': 'General Energy Level', 'denoms': [{'units': 'Participants', 'counts': [{'value': '127', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '8', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '8', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '82', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '21', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '8', 'groupId': 'OG000'}]}]}, {'title': 'Sleep', 'denoms': [{'units': 'Participants', 'counts': [{'value': '128', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '11', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '11', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '95', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '9', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}]}, {'title': 'Other', 'denoms': [{'units': 'Participants', 'counts': [{'value': '31', 'groupId': 'OG000'}]}], 'categories': [{'title': 'Much better', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': 'Slightly better', 'measurements': [{'value': '2', 'groupId': 'OG000'}]}, {'title': 'Unchanged', 'measurements': [{'value': '12', 'groupId': 'OG000'}]}, {'title': 'Slightly worse', 'measurements': [{'value': '8', 'groupId': 'OG000'}]}, {'title': 'Much worse', 'measurements': [{'value': '7', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Baseline, Week 144', 'description': "Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 144), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much better), 2 (slightly better), 3 (unchanged), 4 (slightly worse), or 5 (much worse).", 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Number analyzed 'signifies participants evaluable for specified categories."}, {'type': 'SECONDARY', 'title': 'Ataluren Plasma Concentration', 'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'title': 'Week 12', 'denoms': [{'units': 'Participants', 'counts': [{'value': '210', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.5231', 'spread': '5.08583', 'groupId': 'OG000'}]}]}, {'title': 'Week 24', 'denoms': [{'units': 'Participants', 'counts': [{'value': '211', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '4.4817', 'spread': '5.65883', 'groupId': 'OG000'}]}]}, {'title': 'Week 36', 'denoms': [{'units': 'Participants', 'counts': [{'value': '205', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.1551', 'spread': '5.67555', 'groupId': 'OG000'}]}]}, {'title': 'Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '201', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.2221', 'spread': '5.32846', 'groupId': 'OG000'}]}]}, {'title': 'Week 60', 'denoms': [{'units': 'Participants', 'counts': [{'value': '191', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.0396', 'spread': '5.07812', 'groupId': 'OG000'}]}]}, {'title': 'Week 72', 'denoms': [{'units': 'Participants', 'counts': [{'value': '180', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.0677', 'spread': '6.05912', 'groupId': 'OG000'}]}]}, {'title': 'Week 84', 'denoms': [{'units': 'Participants', 'counts': [{'value': '171', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.4673', 'spread': '5.15083', 'groupId': 'OG000'}]}]}, {'title': 'Week 96', 'denoms': [{'units': 'Participants', 'counts': [{'value': '160', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.8870', 'spread': '5.90954', 'groupId': 'OG000'}]}]}, {'title': 'Week 108', 'denoms': [{'units': 'Participants', 'counts': [{'value': '133', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.5905', 'spread': '6.48016', 'groupId': 'OG000'}]}]}, {'title': 'Week 120', 'denoms': [{'units': 'Participants', 'counts': [{'value': '119', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.2406', 'spread': '5.69648', 'groupId': 'OG000'}]}]}, {'title': 'Week 132', 'denoms': [{'units': 'Participants', 'counts': [{'value': '94', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '6.6729', 'spread': '7.03882', 'groupId': 'OG000'}]}]}, {'title': 'Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '66', 'groupId': 'OG000'}]}], 'categories': [{'measurements': [{'value': '5.3898', 'spread': '6.28487', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Pre-dose at Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144', 'description': 'Pre-dose ataluren plasma concentrations prior to morning ataluren administration at each clinic visit was assessed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method.', 'unitOfMeasure': 'micrograms/milliliter (μg/mL)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Number analyzed' signifies participants evaluable for this outcome measure at specified timepoints."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Systolic and Diastolic Blood Pressure at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'title': 'Systolic blood pressure', 'categories': [{'measurements': [{'value': '0.8', 'spread': '11.86', 'groupId': 'OG000'}]}]}, {'title': 'Diastolic blood pressure', 'categories': [{'measurements': [{'value': '1.3', 'spread': '11.60', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'Blood pressure determination was performed with the participant in a sitting position after a 5-minute rest.', 'unitOfMeasure': 'mmHg', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Pulse Rate at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.9', 'spread': '13.00', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'description': 'Pulse rate determination was performed with the participant in a sitting position after a 5-minute rest.', 'unitOfMeasure': 'beats/minute', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Body Temperature at Week 144', 'denoms': [{'units': 'Participants', 'counts': [{'value': '68', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '0.05', 'spread': '0.545', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Week 144', 'unitOfMeasure': 'degrees centigrade', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': "AT population included all participants who received at least 1 dose of ataluren treatment in this extension study. Here, 'Overall number of participants analyzed' = participants who had both baseline and post-baseline value of this parameter at specified timepoint."}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '219'}]}, {'type': 'As-treated Population', 'comment': 'All participants who received at least 1 dose of study drug.', 'achievements': [{'groupId': 'FG000', 'numSubjects': '218'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '68'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '151'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Investigator decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}, {'type': 'Study termination', 'reasons': [{'groupId': 'FG000', 'numSubjects': '6'}]}, {'type': 'Enrolled but not treated', 'reasons': [{'groupId': 'FG000', 'numSubjects': '1'}]}, {'type': 'Transfer for compassionate use medicine', 'reasons': [{'groupId': 'FG000', 'numSubjects': '4'}]}, {'type': 'Move to medical need program', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}]}, {'type': 'Entered in to other study', 'reasons': [{'groupId': 'FG000', 'numSubjects': '34'}]}, {'type': 'Switched to commercial supply', 'reasons': [{'groupId': 'FG000', 'numSubjects': '88'}]}, {'type': 'Other than specified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}]}]}], 'recruitmentDetails': 'All participants who successfully completed the double-blind, placebo-controlled Phase 3 study (PTC124-GD-020-DMD \\[NCT01826487\\]) were screened for this open-label extension study.', 'preAssignmentDetails': 'A total of 221 participants completed the double-blind Phase 3 Study PTC124-GD-020-DMD. Of the 221 participants who completed Study PTC124-GD-020-DMD, 219 participants were enrolled in this open-label extension study and 218 were treated.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Ataluren', 'description': 'Participants received ataluren suspension orally TID, 10 mg/kg at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '9.9', 'spread': '1.78', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '218', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '218', 'groupId': 'BG000'}]}], 'categories': [{'title': 'White', 'measurements': [{'value': '169', 'groupId': 'BG000'}]}, {'title': 'Black', 'measurements': [{'value': '2', 'groupId': 'BG000'}]}, {'title': 'Asian', 'measurements': [{'value': '13', 'groupId': 'BG000'}]}, {'title': 'Hispanic', 'measurements': [{'value': '12', 'groupId': 'BG000'}]}, {'title': 'Other', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': 'Missing', 'measurements': [{'value': '14', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': '6 Minute Walk Distance (6MWD)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '198', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '349.885', 'spread': '105.7913', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'The 6MWD test was performed in a 30 meters long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.', 'unitOfMeasure': 'meters', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'This baseline characteristic was analyzed on ambulatory participants only.'}, {'title': 'Time to Stand From Supine Position', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '199', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '13.0', 'spread': '10.34', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'seconds', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Time to Walk/Run 10 Meters', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '215', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '9.31', 'spread': '7.388', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'seconds', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Time to Climb 4 Stairs', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '203', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '9.03', 'spread': '8.939', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'seconds', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Time to Descend 4 Stairs', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '204', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '7.52', 'spread': '8.401', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'seconds', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'North Star Ambulatory Assessment (NSAA) Total Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '195', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '20.73', 'spread': '8.513', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'NSAA comprised tests of 17 abilities, such as ability to stand, rise from floor, get from lying to sitting, get from sitting to standing, raise one\'s head, stand on one\'s heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." Sum of these 17 scores was reported as ordinal total score, which can be transformed to a linear total score from 0 (worst) to 100 (best).', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': 'This baseline parameter was analyzed on ambulatory participants with a baseline value.'}, {'title': 'Performance Upper Limb (PUL) Total Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '210', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '68.7', 'spread': '4.61', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into shoulder (4 items), elbow (9 items), and distal (8 items) level dimensions. Scoring options per item may vary from 0-1 and 0-6, according to performance, with higher values corresponding to better performance. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for elbow level, and 24 for distal level. Total score was calculated by adding 3 level scores, with a maximum global score of 74 (total score range = 0-74). Higher score = better outcome.', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Percent Predicted Forced Vital Capacity (FVC)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '210', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '59.42', 'spread': '13.843', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'FVC is a standard pulmonary function test. FVC was defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Percent predicted FVC (in %) = \\[(observed FVC)/(predicted FVC)\\]\\*100.', 'unitOfMeasure': 'percent predicted FVC', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '209', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '53.76', 'spread': '12.870', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'FEV1 is a standard pulmonary function test. FEV1 was defined as the volume of air that can forcibly be blown out in 1 second, after full inspiration in the upright position, measured in liters. Percent predicted FEV1 (in %) = \\[(observed FEV1)/(predicted FEV1)\\]\\*100.', 'unitOfMeasure': 'percent predicted FEV1', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Peak Expiratory Flow (PEF)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '213', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '3.36', 'spread': '1.055', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'PEF was defined as the maximum airflow during a forced expiration beginning with the lungs fully inflated.', 'unitOfMeasure': 'liters/second (L/sec)', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Peak Cough Flow (PCF)', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '194', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '3.30', 'spread': '1.139', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': "PCF measures an individual's maximum speed of expiration during cough.", 'unitOfMeasure': 'L/sec', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Pediatric Outcomes Data Collection Instrument (PODCI) Transfers/Basic Mobility Score', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '216', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '74.6', 'spread': '23.66', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'description': 'PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity \\& Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. Following PODCI domains were prespecified in protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Each domain was scored from 0 (poor outcome/worse health) to 100 (the highest level of functioning \\& least pain).', 'unitOfMeasure': 'units on a scale', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Blood Pressure', 'classes': [{'title': 'Systolic blood pressure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '217', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '106.3', 'spread': '10.72', 'groupId': 'BG000'}]}]}, {'title': 'Diastolic blood pressure', 'denoms': [{'units': 'Participants', 'counts': [{'value': '217', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '68.6', 'spread': '11.01', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'millimeters of mercury (mm Hg)', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Pulse Rate', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '217', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '97.0', 'spread': '13.31', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'beats/minute', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}, {'title': 'Body Temperature', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '217', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '36.47', 'spread': '0.453', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'degrees centigrade', 'dispersionType': 'STANDARD_DEVIATION', 'populationDescription': "'Number analyzed' signifies participants of as-treated population analyzed for this parameter."}], 'populationDescription': 'As Treated (AT) population included all participants who received at least 1 dose of ataluren treatment in this extension study.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2015-11-19', 'size': 8281830, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-07-17T15:17', 'hasProtocol': True}, {'date': '2019-01-23', 'size': 2051091, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-07-17T15:17', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 219}}, 'statusModule': {'whyStopped': 'Sponsor decision due to commercial availability of ataluren.', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2014-03-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-08', 'dispFirstSubmitDate': '2019-04-09', 'completionDateStruct': {'date': '2018-06-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-08-10', 'studyFirstSubmitDate': '2014-03-17', 'dispFirstSubmitQcDate': '2019-04-09', 'resultsFirstSubmitDate': '2020-07-17', 'studyFirstSubmitQcDate': '2014-03-17', 'dispFirstPostDateStruct': {'date': '2019-04-12', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2020-08-11', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2020-08-10', 'studyFirstPostDateStruct': {'date': '2014-03-19', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-08-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-06-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment-Emergent Adverse Events (TEAEs)', 'timeFrame': 'Baseline (Day 1) up to 6 weeks post-treatment (Week 150)', 'description': 'An adverse event (AE): any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of AEs: graded per Common Terminology Criteria for AEs (CTCAE), Version 3.0 as Grade 1 (mild), 2 (moderate), 3 (severe), 4 (life-threatening), 5 (death). Drug-related AEs: AEs with possible, probable, unlikely relationship, or unrelated to study drug. Serious AEs (SAEs): death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: an AE that occurred or worsened in the period extending from first dose of study drug in this study to 6 weeks after last dose of study drug in this study. A summary of other non-serious AEs and all SAEs, regardless of causality is located in Reported AE section.'}, {'measure': 'Number of Participants With Abnormalities in Clinical Laboratory Parameters', 'timeFrame': 'Baseline (Day 1) up to 6 weeks post-treatment (Week 150)', 'description': 'Abnormalities in laboratory variables as pre-defined in protocol for safety-monitoring were: Hepatic (Serum alanine aminotransferase \\[ALT\\]: increase of greater than \\[\\>\\] 150 units/liter \\[U/L\\] with stable or decrease of creatinine kinese \\[CK\\]; Serum glutamyl amino transferase \\[GGT\\] \\[U/L\\]: Grade 2 \\[\\>2.5 - 5.0 \\* upper limit of normal {ULN}\\]), renal (Serum cystatin C miiligrams/liter \\[mg/L\\] \\>1.33 - 2.00 mg/L; Serum blood urea nitrogen \\[UREAN\\] \\[millimoles/liter {mmol/L}\\] greater than or equal to \\[≥\\]1.5 - 3.0 \\* ULN; Urine occult blood: 2+ \\[Small\\], 3+ \\[Moderate\\], 4+ \\[Large\\]), and electrolytes (Serum sodium: low \\[mmol/L\\], Grade 3-4 \\[less than {\\<}130 mmol/L\\]; serum potassium: high \\[mmol/L\\], Grade 3-4 \\[\\>6.0 mmol/L\\]; and Serum bicarbonate \\[mmol/L\\]: Grade 2 \\[\\<16 - 11 mmol/L\\]).'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in 6MWD at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'The 6MWD test was performed in a 30 meters long flat corridor, where the participant was instructed to walk as far as possible, back and forth around two cones, with the permission to slow down, rest, or stop if needed. Ambulation was assessed via the 6MWD test following standardized procedures by measuring the 6MWD in meters. Participants were not permitted to use assistive devices (walker, long leg braces, or short leg braces) during the 6MWD test.'}, {'measure': 'Change From Baseline in Time to Stand From Supine Position at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.'}, {'measure': 'Change From Baseline in Time to Walk/Run 10 Meters at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.'}, {'measure': 'Change From Baseline in Time to Climb 4 Stairs at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.'}, {'measure': 'Change From Baseline in Time to Descend 4 Stairs at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'If the time taken to perform this test exceeded 30 seconds or if a participant could not perform this test due to disease progression, a value of 30 seconds was used.'}, {'measure': 'Change From Baseline in Physical Function Total Score as Measured by NSAA at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'Physical function was assessed via the NSAA, a functional scale specifically designed for ambulant Duchenne muscular dystrophy (DMD) participants. The assessment comprised tests for 17 abilities of a participant, such as ability to stand, rise from the floor, get from lying to sitting, get from sitting to standing, raise one\'s head, stand on one\'s heels, hop, jump, and run. For each activity, a score of 0, 1, or 2 was recorded, with 0 = "unable to achieve independently," 1 = "modified method but achieves goal independent of physical assistance from another," or 2 = "normal- achieves goal without any assistance." The sum of these scores (except for \'raise one\'s head\' activity score) was reported as the ordinal total score, which was transformed to a linear total score ranging from 0 (worst) to 100 (best). Participants with confirmed loss of ambulation at a particular visit were assigned a score of 0.'}, {'measure': 'Change From Baseline in PUL Total Score at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'The PUL was used to assess motor performance of the upper limb. The PUL scale includes 22 items; an entry item defining the starting functional level, and 21 items subdivided into shoulder level (4 items), elbow level (9 items), and distal level (8 items) dimensions. Scoring options per item may not be uniform and may vary from 0-1 and 0-6, according to the performance, with higher values corresponding to better performance. Each dimension was scored separately with a maximum score of 16 for shoulder level, 34 for elbow level, and 24 for distal level. Total score was calculated by adding the 3 level scores, with a maximum global score of 74 (total score range = 0-74). Higher score = better outcome.'}, {'measure': 'Change From Baseline in Percent Predicted FVC as Measured by Spirometry at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'FVC is a standard pulmonary function test. FVC was defined as the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Percent predicted FVC (in %) = \\[(observed FVC)/(predicted FVC)\\]\\*100.'}, {'measure': 'Change From Baseline in Percent Predicted FEV1 as Measured by Spirometry at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'FEV1 is a standard pulmonary function test. FEV1 was defined as the volume of air that can forcibly be blown out in 1 second, after full inspiration in the upright position, measured in liters. Percent predicted FEV1 (in %) = \\[(observed FEV1)/(predicted FEV1)\\]\\*100.'}, {'measure': 'Change From Baseline in PEF as Measured by Spirometry at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'PEF was defined as the maximum airflow during a forced expiration beginning with the lungs fully inflated.'}, {'measure': 'Change From Baseline in PCF as Measured by Spirometry at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': "PCF measures an individual's maximum speed of expiration during cough."}, {'measure': 'Change From Baseline in PODCI Transfers/Basic Mobility Score at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'Changes in health-related quality of life (HRQL) were measured via the PODCI questionnaire that has been shown to correlate with disease progression and clinical outcome measures in DMD. PODCI includes a Global Functioning Scale and 5 core scales: Upper Extremity and Physical Function,Transfer/Basic Mobility, Sports/Physical Functioning, Pain/Comfort,and Happiness. The following PODCI domain was prespecified in the protocol for analysis:Transfers/Basic Mobility domain assesses difficulty experienced in performing routine motor activities in daily life. Each domain was scored from 0 to 100, with 0 representing a poor outcome/worse health, while 100 representing the highest level of functioning and least pain.'}, {'measure': 'Number of Participants With Change From Baseline in Activities of Daily Living and Disease Status at Week 144, as Assessed by a Standardized Survey Administered by Site Personnel', 'timeFrame': 'Baseline, Week 144', 'description': "Changes in activities of daily living and disease symptoms were captured via a DMD-specific survey administered by Site personnel. At screening or baseline, the participant and/or parent/caregiver were asked to identify any activities of daily living (for example, ambulation, balance, personal hygiene/grooming, dressing and undressing, self-feeding, using the bathroom, handwriting, school performance, behavior or energy level) or symptoms that were affected by the participant's DMD. At post-baseline visit (Week 144), the same participant and/or parent/caregiver was asked to describe any changes from baseline in those activities of daily living/symptoms, within the following categories: physical functioning; general energy level; cognition/school function; emotional/social functioning; and sleep. Changes from baseline were reported on a 5-point Likert scale: 1 (much better), 2 (slightly better), 3 (unchanged), 4 (slightly worse), or 5 (much worse)."}, {'measure': 'Ataluren Plasma Concentration', 'timeFrame': 'Pre-dose at Weeks 12, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, and 144', 'description': 'Pre-dose ataluren plasma concentrations prior to morning ataluren administration at each clinic visit was assessed using a validated high performance liquid chromatography with tandem mass spectrometry (HPLC/MS-MS) method.'}, {'measure': 'Change From Baseline in Systolic and Diastolic Blood Pressure at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'Blood pressure determination was performed with the participant in a sitting position after a 5-minute rest.'}, {'measure': 'Change From Baseline in Pulse Rate at Week 144', 'timeFrame': 'Baseline, Week 144', 'description': 'Pulse rate determination was performed with the participant in a sitting position after a 5-minute rest.'}, {'measure': 'Change From Baseline in Body Temperature at Week 144', 'timeFrame': 'Baseline, Week 144'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Duchenne muscular dystrophy', 'Dystrophinopathy', 'Nonsense mutation', 'Premature stop codon', 'Becker muscular dystrophy', 'DMD/BMD', 'PTC124', 'Ataluren'], 'conditions': ['Muscular Dystrophy, Duchenne', 'Muscular Dystrophies', 'Muscular Disorders, Atrophic', 'Muscular Diseases', 'Musculoskeletal Diseases', 'Neuromuscular Diseases', 'Nervous System Diseases', 'Genetic Diseases, X-Linked', 'Genetic Diseases, Inborn']}, 'referencesModule': {'references': [{'pmid': '30135256', 'type': 'DERIVED', 'citation': 'Thangarajh M, Elfring GL, Trifillis P, McIntosh J, Peltz SW; Ataluren Phase 2b Study Group. The relationship between deficit in digit span and genotype in nonsense mutation Duchenne muscular dystrophy. Neurology. 2018 Sep 25;91(13):e1215-e1219. doi: 10.1212/WNL.0000000000006245. Epub 2018 Aug 22.'}], 'seeAlsoLinks': [{'url': 'http://www.ptcbio.com', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': 'The primary objective of this study is to obtain long term safety data of ataluren in male participants with nonsense mutation dystrophinopathy (who participated and completed a previous Phase 3 study of ataluren \\[PTC124-GD-020-DMD {NCT01826487}\\]) to augment the overall safety database. Screening and baseline procedures are structured to avoid a gap in treatment between the double-blind study (PTC124-GD-020-DMD) and this extension study.\n\nThis study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is discontinued.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD'], 'maximumAge': '15 Years', 'minimumAge': '7 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Completion of study treatment in the previous Phase 3, double-blind study (PTC124-GD-020-DMD).\n* Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether 1 or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.\n* In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the period of study drug administration and 6-week follow-up period.\n* Willingness and ability to comply with scheduled visits, ataluren administration plan, study procedures, laboratory tests, and study restrictions.\n\nExclusion Criteria:\n\n* Known hypersensitivity to any of the ingredients or excipients of the study drug (Litesse® UltraTM \\[refined polydextrose\\], polyethylene glycol 3350, Lutrol® micro F127 \\[poloxamer 407\\], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P \\[colloidal silica\\], and magnesium stearate).\n* Ongoing participation in any other therapeutic clinical trial.\n* Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results."}, 'identificationModule': {'nctId': 'NCT02090959', 'briefTitle': 'An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy', 'organization': {'class': 'INDUSTRY', 'fullName': 'PTC Therapeutics'}, 'officialTitle': 'A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy', 'orgStudyIdInfo': {'id': 'PTC124-GD-020e-DMD'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Ataluren', 'description': 'Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for up to 144 weeks.', 'interventionNames': ['Drug: Ataluren']}], 'interventions': [{'name': 'Ataluren', 'type': 'DRUG', 'otherNames': ['PTC124'], 'description': 'Ataluren will be administered per the dose and schedule specified in the arm.', 'armGroupLabels': ['Ataluren']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'UC Davis Medical Center', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University Medical Center', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital Colorado - 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Childhood Cancer and Blood Disorders", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': "The Children's Hospital at Westmead", 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '3052', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': "The Royal Children's Hospital", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': '3000', 'city': 'Leuven', 'country': 'Belgium', 'facility': 'UZ Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'zip': '21.941-912', 'city': 'Rio de Janeiro', 'country': 'Brazil', 'facility': 'Universidade Federal do Rio de Janeiro - Instituto de Puericultura e Pediatria Martagao Gesteira', 'geoPoint': {'lat': -22.90642, 'lon': -43.18223}}, {'zip': '05403-900', 'city': 'São Paulo', 'country': 'Brazil', 'facility': 'Sao Paulo University -HC/FMUSP', 'geoPoint': {'lat': -23.5475, 'lon': -46.63611}}, {'zip': '1431', 'city': 'Sofia', 'country': 'Bulgaria', 'facility': 'Aleksandrovska Hospital', 'geoPoint': {'lat': 42.69751, 'lon': 23.32415}}, {'zip': 'T3B 6A8', 'city': 'Calgary', 'state': 'Alberta', 'country': 'Canada', 'facility': "Alberta Children's Hospital", 'geoPoint': {'lat': 51.05011, 'lon': -114.08529}}, {'zip': 'V6H 3V4', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "British Columbia Children's Hospital", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'N6C 2RC', 'city': 'London', 'state': 'Ontario', 'country': 'Canada', 'facility': "Children's Hospital of Western Ontario", 'geoPoint': {'lat': 42.98339, 'lon': -81.23304}}, {'city': 'Santiago', 'state': 'Santiago Metropolitan', 'country': 'Chile', 'facility': 'Hospital Luis Calvo Mackenna', 'geoPoint': {'lat': -33.45694, 'lon': -70.64827}}, {'zip': '8330073', 'city': 'Santiago', 'country': 'Chile', 'facility': 'Hospital Clinico Universidad Catolica', 'geoPoint': {'lat': -33.45694, 'lon': -70.64827}}, {'zip': '635 00', 'city': 'Brno', 'country': 'Czechia', 'facility': 'University Hospital Brno', 'geoPoint': {'lat': 49.19522, 'lon': 16.60796}}, {'zip': '150 06', 'city': 'Prague', 'country': 'Czechia', 'facility': 'Motol University Hospital', 'geoPoint': {'lat': 50.08804, 'lon': 14.42076}}, {'zip': '13385', 'city': 'Marseille', 'country': 'France', 'facility': 'Hospital de la Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'zip': '44093', 'city': 'Nantes', 'country': 'France', 'facility': 'CHU de Nantes', 'geoPoint': {'lat': 47.21725, 'lon': -1.55336}}, {'zip': '75015', 'city': 'Paris', 'country': 'France', 'facility': 'Hopital Necker - Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '75651', 'city': 'Paris', 'country': 'France', 'facility': 'Groupe Hospitalier Pitie-Salpetriere', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '45122', 'city': 'Essen', 'country': 'Germany', 'facility': 'Universitaetsklinikum Essen', 'geoPoint': {'lat': 51.45657, 'lon': 7.01228}}, {'zip': '79106', 'city': 'Freiburg im Breisgau', 'country': 'Germany', 'facility': 'University Medical Center Freiburg', 'geoPoint': {'lat': 47.9959, 'lon': 7.85222}}, {'zip': '80337', 'city': 'München', 'country': 'Germany', 'facility': 'Universitat Munchen - von Haunersche Kinder Clinic', 'geoPoint': {'lat': 51.60698, 'lon': 13.31243}}, {'zip': '91240', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Hadassah University Hospital', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'zip': '20122', 'city': 'Milan', 'country': 'Italy', 'facility': 'Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico di Milano', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '00165', 'city': 'Rome', 'country': 'Italy', 'facility': 'Bambino Gesu Hospital', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'zip': '00168', 'city': 'Rome', 'country': 'Italy', 'facility': 'U.O. 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