Ignite Creation Date:
2025-12-25 @ 1:21 AM
Ignite Modification Date:
2026-01-04 @ 8:09 PM
Study NCT ID:
NCT02960893
Status:
COMPLETED
Last Update Posted:
2025-10-08
First Post:
2016-11-04
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Trial in Adult Participants With Spinocerebellar Ataxia (SCA)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020754', 'term': 'Spinocerebellar Ataxias'}], 'ancestors': [{'id': 'D002524', 'term': 'Cerebellar Ataxia'}, {'id': 'D002526', 'term': 'Cerebellar Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D013132', 'term': 'Spinocerebellar Degenerations'}, {'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D001259', 'term': 'Ataxia'}, {'id': 'D020820', 'term': 'Dyskinesias'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'clinicaltrials@biohavenpharma.com', 'phone': '203-404-0410', 'title': 'Chief Medical Officer', 'organization': 'Biohaven Pharmaceuticals'}, 'certainAgreement': {'restrictionType': 'LTE60', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 348 weeks after last randomization participant was enrolled)', 'description': 'Safety Analysis Set: All participants who received at least one dose of study drug during the Randomization Phase/OLE Phase\n\nMedDRA versions used: Randomization phase: v19.0, OLE Phase: v20.0 and v27.0', 'eventGroups': [{'id': 'EG000', 'title': 'Troriluzole - Randomization Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.', 'otherNumAtRisk': 71, 'deathsNumAtRisk': 71, 'otherNumAffected': 22, 'seriousNumAtRisk': 71, 'deathsNumAffected': 0, 'seriousNumAffected': 4}, {'id': 'EG001', 'title': 'Placebo - Randomization Phase', 'description': 'Participants received matching placebo capsules orally QD for 8 weeks.', 'otherNumAtRisk': 70, 'deathsNumAtRisk': 70, 'otherNumAffected': 18, 'seriousNumAtRisk': 70, 'deathsNumAffected': 0, 'seriousNumAffected': 1}, {'id': 'EG002', 'title': 'Troriluzole/Troriluzole - OLE Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.', 'otherNumAtRisk': 64, 'deathsNumAtRisk': 64, 'otherNumAffected': 48, 'seriousNumAtRisk': 64, 'deathsNumAffected': 2, 'seriousNumAffected': 11}, {'id': 'EG003', 'title': 'Placebo/Troriluzole - OLE Phase', 'description': 'Participants who received placebo during randomization phase, received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.', 'otherNumAtRisk': 67, 'deathsNumAtRisk': 67, 'otherNumAffected': 50, 'seriousNumAtRisk': 67, 'deathsNumAffected': 1, 'seriousNumAffected': 9}], 'otherEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 9}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 7}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 8}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 10}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 21}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 19}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Arthralgia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 9}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 10}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 7}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 5}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Muscle spasms', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 2}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 8}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 10}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 14}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 4}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 4}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 12}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 9}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Balance disorder', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 7}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 6}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 7}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Urinary tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 9}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Contusion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 10}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Ligament sprain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 5}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Skin laceration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 5}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Skin abrasion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 5}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 9}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 9}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Pain in extremity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 3}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 6}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 6}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Abdominal discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Gastrooesophageal reflux disease', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Insomnia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 7}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Anxiety', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 5}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Depression', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 4}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Suicidal ideation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 3}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 6}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Oropharyngeal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 3}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 4}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 5}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Vertigo', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 5}], 'organSystem': 'Ear and labyrinth disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}], 'seriousEvents': [{'term': 'Pneumonia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 2}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Asthenia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Inflammation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Chest discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Atrial fibrillation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Blood creatine phosphokinase increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Dehydration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Back pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Cerebral infarction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Syncope', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Suicidal ideation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Hospitalisation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Hypertension', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 1}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Vascular disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Pneumonia aspiration', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 2}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'COVID-19', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Sepsis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Tibia fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 3}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Fibula fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 2}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Ankle fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Concussion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Fall', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Hip fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Joint dislocation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Upper limb fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Colitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Diarrhoea haemorrhagic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Dysphagia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Gastrointestinal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Lower gastrointestinal haemorrhage', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Acute myocardial infarction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Myocardial infarction', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Cardiac disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Adult failure to thrive', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Muscular weakness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Adenocarcinoma pancreas', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Pancreatic carcinoma metastatic', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Neoplasms benign, malignant and unspecified (incl cysts and polyps)', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Transient global amnesia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Mental status changes', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'Psychiatric disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Cholecystitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 1}], 'organSystem': 'Hepatobiliary disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Respiratory failure', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}, {'term': 'Ankle operation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 71, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 70, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 64, 'numAffected': 1}, {'groupId': 'EG003', 'numAtRisk': 67, 'numAffected': 0}], 'organSystem': 'Surgical and medical procedures', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA (19, 20, 27)'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'OG000'}, {'value': '68', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Troriluzole - Randomization Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.'}, {'id': 'OG001', 'title': 'Placebo - Randomization Phase', 'description': 'Participants received matching placebo capsules orally QD for 8 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.810', 'spread': '1.7653', 'groupId': 'OG000'}, {'value': '-1.059', 'spread': '2.3221', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.519', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '0.2', 'ciLowerLimit': '-0.5', 'ciUpperLimit': '0.9', 'pValueComment': 'Significance was evaluated at a 2-sided alpha level of 0.05', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '0.4', 'statisticalMethod': 'Mixed Model with Repeated Measures', 'nonInferiorityType': 'SUPERIORITY', 'statisticalComment': 'Fixed effects: treatment, baseline (BL) gait severity, visit, treatment-by-visit interaction; Covariate: BL Total SARA score; Random effect: subject'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, Randomization Phase Week 8', 'description': 'The severity of ataxia was assessed with the SARA, an 8-item clinical rating scale from 0 (no ataxia) to 40 (most severe ataxia). The total score was derived as the sum of the individual items, which included gait (0-8), stance (0-6), sitting (0-4), speech disturbance (0-6), finger chase (0-4), nose-finger test (0-4), fast alternating hand movements (0-4), and heel-shin slide (0-4). Since the finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide were repeated on both the right and left side, the average of the right and left side assessments was used to derive the total score. A negative change in score shows improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set (FAS): All randomized participants with at least one dose of study drug and a baseline and post-baseline Total SARA score during the Randomization Phase. Here, number of participants analyzed signifies the number of evaluable participants with change from baseline in Total SARA score at Week 8.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '71', 'groupId': 'OG000'}, {'value': '70', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Troriluzole - Randomization Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 8 weeks.'}, {'id': 'OG001', 'title': 'Placebo - Randomization Phase', 'description': 'Participants received matching placebo capsules orally QD for 8 weeks.'}], 'classes': [{'title': 'Deaths', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'Discontinued due to AEs', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs', 'categories': [{'measurements': [{'value': '40', 'groupId': 'OG000'}, {'value': '35', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks)', 'description': 'An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Analysis Set (Randomization Phase): All participants who received at least one dose of study drug during the Randomization Phase.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '64', 'groupId': 'OG000'}, {'value': '67', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Troriluzole/Troriluzole - OLE Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 336 weeks.'}, {'id': 'OG001', 'title': 'Placebo/Troriluzole - OLE Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 336 weeks.'}], 'classes': [{'title': 'Deaths', 'categories': [{'measurements': [{'value': '2', 'groupId': 'OG000'}, {'value': '1', 'groupId': 'OG001'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '11', 'groupId': 'OG000'}, {'value': '9', 'groupId': 'OG001'}]}]}, {'title': 'Discontinued due to AEs', 'categories': [{'measurements': [{'value': '9', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'TEAEs', 'categories': [{'measurements': [{'value': '60', 'groupId': 'OG000'}, {'value': '59', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 weeks)', 'description': 'An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Safety Analysis Set (OLE Phase): All participants who received at least one dose of study drug during the OLE Phase.'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Received At Least One Dose of Troriluzole in the Randomization Phase or OLE Phase With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs', 'denoms': [{'units': 'Participants', 'counts': [{'value': '138', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Troriluzole - Overall', 'description': 'Participants received at least one dose of Troriluzole 140 mg capsule orally QD during the Randomization Phase or OLE Phase.'}], 'classes': [{'title': 'Deaths', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}]}]}, {'title': 'SAEs', 'categories': [{'measurements': [{'value': '23', 'groupId': 'OG000'}]}]}, {'title': 'Discontinued due to AEs', 'categories': [{'measurements': [{'value': '19', 'groupId': 'OG000'}]}]}, {'title': 'TEAEs', 'categories': [{'measurements': [{'value': '126', 'groupId': 'OG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 348 weeks after last randomization participant was enrolled)', 'description': 'An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'All participants who received at least one dose of Troriluzole during the Randomization Phase or OLE Phase.'}, {'type': 'SECONDARY', 'title': 'Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase', 'denoms': [{'units': 'Participants', 'counts': [{'value': '63', 'groupId': 'OG000'}, {'value': '66', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Troriluzole - Randomization Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally once daily QD for 8 weeks.'}, {'id': 'OG001', 'title': 'Placebo - Randomization Phase', 'description': 'Participants received matching placebo capsules orally QD for 8 weeks.'}], 'classes': [{'title': 'No Change', 'categories': [{'measurements': [{'value': '31', 'groupId': 'OG000'}, {'value': '27', 'groupId': 'OG001'}]}]}, {'title': 'Almost the same', 'categories': [{'measurements': [{'value': '15', 'groupId': 'OG000'}, {'value': '15', 'groupId': 'OG001'}]}]}, {'title': 'A little better', 'categories': [{'measurements': [{'value': '7', 'groupId': 'OG000'}, {'value': '11', 'groupId': 'OG001'}]}]}, {'title': 'Somewhat better', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '2', 'groupId': 'OG001'}]}]}, {'title': 'Moderately better', 'categories': [{'measurements': [{'value': '3', 'groupId': 'OG000'}, {'value': '7', 'groupId': 'OG001'}]}]}, {'title': 'Better', 'categories': [{'measurements': [{'value': '4', 'groupId': 'OG000'}, {'value': '4', 'groupId': 'OG001'}]}]}, {'title': 'A great deal better', 'categories': [{'measurements': [{'value': '0', 'groupId': 'OG000'}, {'value': '0', 'groupId': 'OG001'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Randomization Phase Week 8', 'description': 'PGI-C is a patient self-reported global index scale that was used to rate the response of a condition to therapy. Participants rated their impression of benefit based on the following 7 categories: No change (or condition has gotten worse); Almost the same, hardly any change at all; A little better, but no noticeable change; Somewhat better, but the change has not made any real difference; Moderately better, and a slight but noticeable change; Better and a definitive improvement that has made a real and worthwhile difference; A great deal better and a considerable improvement that has made all the difference.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set (FAS): All randomized participants with at least one dose of study drug and a baseline and post-baseline Total SARA score during the Randomization Phase. Here, number of participants analyzed signifies the number of evaluable participants for PGI-C index at Week 8.'}, {'type': 'OTHER_PRE_SPECIFIED', 'title': 'Change From Randomization Baseline in Total Score on the SARA at Extension Phase Week 48', 'denoms': [{'units': 'Participants', 'counts': [{'value': '96', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Troriluzole - Extension Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 48 weeks.'}], 'classes': [{'categories': [{'measurements': [{'value': '-0.42', 'spread': '2.522', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Randomization Baseline, Extension Phase Week 48', 'description': 'The severity of ataxia was assessed with the SARA, an 8-item clinical rating scale from 0 (no ataxia) to 40 (most severe ataxia). The total score was derived as the sum of the individual items, which included gait (0-8), stance (0-6), sitting (0-4), speech disturbance (0-6), finger chase (0-4), nose-finger test (0-4), fast alternating hand movements (0-4), and heel-shin slide (0-4). Since the finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide were repeated on both the right and left side, the average of the right and left side assessments was used to derive the total score. A negative change in score shows improvement.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set (FAS): All randomized participants with at least one dose of study drug and a baseline and post-baseline Total SARA score during the Randomization Phase. Here, number of participants analyzed signifies the number of evaluable participants with change from Randomization baseline in Total SARA score at Week 48.'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Troriluzole - Randomization Phase', 'description': 'Participants received Troriluzole 140 milligrams (mg) capsules orally once daily (QD) for 8 weeks.'}, {'id': 'FG001', 'title': 'Placebo - Randomization Phase', 'description': 'Participants received matching placebo capsules orally QD for 8 weeks.'}, {'id': 'FG002', 'title': 'Troriluzole/Troriluzole - Open Label Extension (OLE) Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.'}, {'id': 'FG003', 'title': 'Placebo/Troriluzole - OLE Phase', 'description': 'Participants who received placebo during randomization phase, received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.'}], 'periods': [{'title': 'Randomization Phase (8 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '71'}, {'groupId': 'FG001', 'numSubjects': '70'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'COMPLETED', 'comment': 'Completed the Randomization Phase', 'achievements': [{'groupId': 'FG000', 'numSubjects': '64'}, {'groupId': 'FG001', 'numSubjects': '68'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '7'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '5'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Withdrawal by Participant', 'reasons': [{'groupId': 'FG000', 'numSubjects': '2'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}, {'type': 'Reason Not Specified', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}, {'groupId': 'FG002', 'numSubjects': '0'}, {'groupId': 'FG003', 'numSubjects': '0'}]}]}, {'title': 'Open Label Extension Phase (336 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '64'}, {'comment': 'One participant in placebo arm who completed Randomization Phase did not continue to OLE Phase', 'groupId': 'FG003', 'numSubjects': '67'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '28'}, {'groupId': 'FG003', 'numSubjects': '36'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '36'}, {'groupId': 'FG003', 'numSubjects': '31'}]}], 'dropWithdraws': [{'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '4'}, {'groupId': 'FG003', 'numSubjects': '4'}]}, {'type': 'Death', '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': '3'}, {'groupId': 'FG003', 'numSubjects': '3'}]}, {'type': 'Physician Decision', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '3'}, {'groupId': 'FG003', 'numSubjects': '2'}]}, {'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '19'}, {'groupId': 'FG003', 'numSubjects': '18'}]}, {'type': 'Sponsor Request', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '4'}, {'groupId': 'FG003', 'numSubjects': '2'}]}, {'type': 'Miscellaneous', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}, {'groupId': 'FG002', 'numSubjects': '3'}, {'groupId': 'FG003', 'numSubjects': '1'}]}]}], 'recruitmentDetails': '181 participants were screened, and 141 of these participants were randomized to treatment at 18 sites in the United States.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '71', 'groupId': 'BG000'}, {'value': '70', 'groupId': 'BG001'}, {'value': '141', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Troriluzole - Randomization Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks.'}, {'id': 'BG001', 'title': 'Placebo - Randomization Phase', 'description': 'Placebo - Participants received matching placebo capsules orally QD for 8 weeks.'}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '52.5', 'spread': '15.08', 'groupId': 'BG000'}, {'value': '51.6', 'spread': '13.74', 'groupId': 'BG001'}, {'value': '52.1', 'spread': '14.39', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '35', 'groupId': 'BG000'}, {'value': '37', 'groupId': 'BG001'}, {'value': '72', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '36', 'groupId': 'BG000'}, {'value': '33', 'groupId': 'BG001'}, {'value': '69', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '65', 'groupId': 'BG000'}, {'value': '64', 'groupId': 'BG001'}, {'value': '129', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race (NIH/OMB)', 'classes': [{'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '5', 'groupId': 'BG000'}, {'value': '11', 'groupId': 'BG001'}, {'value': '16', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '4', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '56', 'groupId': 'BG000'}, {'value': '53', 'groupId': 'BG001'}, {'value': '109', 'groupId': 'BG002'}]}, {'title': 'More than one race', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Spinocerebellar Ataxia (SCA) Genotype', 'classes': [{'title': 'SCA1', 'categories': [{'measurements': [{'value': '17', 'groupId': 'BG000'}, {'value': '18', 'groupId': 'BG001'}, {'value': '35', 'groupId': 'BG002'}]}]}, {'title': 'SCA2', 'categories': [{'measurements': [{'value': '19', 'groupId': 'BG000'}, {'value': '18', 'groupId': 'BG001'}, {'value': '37', 'groupId': 'BG002'}]}]}, {'title': 'SCA3', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '14', 'groupId': 'BG002'}]}]}, {'title': 'SCA6', 'categories': [{'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '15', 'groupId': 'BG001'}, {'value': '31', 'groupId': 'BG002'}]}]}, {'title': 'SCA7', 'categories': [{'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '5', 'groupId': 'BG001'}, {'value': '7', 'groupId': 'BG002'}]}]}, {'title': 'SCA8', 'categories': [{'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '6', 'groupId': 'BG001'}, {'value': '12', 'groupId': 'BG002'}]}]}, {'title': 'SCA10', 'categories': [{'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '2', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'description': 'The Spinocerebellar Ataxia (SCA) data displayed are not grades or stages of disease, but rather the SCA genotypes, which do not correlate with severity of disease.', 'unitOfMeasure': 'Participants'}], 'populationDescription': 'Safety Analysis Set (Randomization Phase): All participants who received at least one dose of study drug during the Randomization Phase.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2020-04-17', 'size': 2286564, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2020-07-24T08:48', 'hasProtocol': True}, {'date': '2020-03-27', 'size': 5901081, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2020-07-24T08:50', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 141}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-12-15', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'nctId': 'NCT06034886', 'statusForNctId': 'AVAILABLE', 'hasExpandedAccess': True}, 'statusVerifiedDate': '2025-09', 'dispFirstSubmitDate': '2018-08-01', 'completionDateStruct': {'date': '2024-09-20', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-09-19', 'studyFirstSubmitDate': '2016-11-04', 'dispFirstSubmitQcDate': '2018-08-06', 'resultsFirstSubmitDate': '2020-07-24', 'studyFirstSubmitQcDate': '2016-11-08', 'dispFirstPostDateStruct': {'date': '2018-08-08', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-10-08', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2020-08-13', 'studyFirstPostDateStruct': {'date': '2016-11-10', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2020-08-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2017-08-18', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change From Randomization Baseline in Total Score on the SARA at Extension Phase Week 48', 'timeFrame': 'Randomization Baseline, Extension Phase Week 48', 'description': 'The severity of ataxia was assessed with the SARA, an 8-item clinical rating scale from 0 (no ataxia) to 40 (most severe ataxia). The total score was derived as the sum of the individual items, which included gait (0-8), stance (0-6), sitting (0-4), speech disturbance (0-6), finger chase (0-4), nose-finger test (0-4), fast alternating hand movements (0-4), and heel-shin slide (0-4). Since the finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide were repeated on both the right and left side, the average of the right and left side assessments was used to derive the total score. A negative change in score shows improvement.'}], 'primaryOutcomes': [{'measure': 'Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8', 'timeFrame': 'Baseline, Randomization Phase Week 8', 'description': 'The severity of ataxia was assessed with the SARA, an 8-item clinical rating scale from 0 (no ataxia) to 40 (most severe ataxia). The total score was derived as the sum of the individual items, which included gait (0-8), stance (0-6), sitting (0-4), speech disturbance (0-6), finger chase (0-4), nose-finger test (0-4), fast alternating hand movements (0-4), and heel-shin slide (0-4). Since the finger chase, nose-finger test, fast alternating hand movements, and heel-shin slide were repeated on both the right and left side, the average of the right and left side assessments was used to derive the total score. A negative change in score shows improvement.'}], 'secondaryOutcomes': [{'measure': 'Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase', 'timeFrame': 'From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks)', 'description': 'An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug.'}, {'measure': 'Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase', 'timeFrame': 'From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 weeks)', 'description': 'An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug.'}, {'measure': 'Number of Participants Who Received At Least One Dose of Troriluzole in the Randomization Phase or OLE Phase With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs', 'timeFrame': 'From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 348 weeks after last randomization participant was enrolled)', 'description': 'An AE was defined as any new untoward medical occurrence or worsening of a pre-existing medical condition, unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. An SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or suspected transmission of an infectious agent, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes. TEAEs were defined as those AEs that developed, worsened, or became serious after the first dose of study drug.'}, {'measure': 'Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase', 'timeFrame': 'Randomization Phase Week 8', 'description': 'PGI-C is a patient self-reported global index scale that was used to rate the response of a condition to therapy. Participants rated their impression of benefit based on the following 7 categories: No change (or condition has gotten worse); Almost the same, hardly any change at all; A little better, but no noticeable change; Somewhat better, but the change has not made any real difference; Moderately better, and a slight but noticeable change; Better and a definitive improvement that has made a real and worthwhile difference; A great deal better and a considerable improvement that has made all the difference.'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Spinocerebellar Ataxia', 'SCA'], 'conditions': ['Spinocerebellar Ataxias', 'Spinocerebellar Ataxia Genotype Type 1', 'Spinocerebellar Ataxia Genotype Type 2', 'Spinocerebellar Ataxia Genotype Type 3', 'Spinocerebellar Ataxia Genotype Type 6', 'Spinocerebellar Ataxia Genotype Type 7', 'Spinocerebellar Ataxia Genotype Type 8', 'Spinocerebellar Ataxia Genotype Type 10']}, 'referencesModule': {'references': [{'pmid': '34115419', 'type': 'DERIVED', 'citation': "Schmahmann JD, Pierce S, MacMore J, L'Italien GJ. Development and Validation of a Patient-Reported Outcome Measure of Ataxia. Mov Disord. 2021 Oct;36(10):2367-2377. doi: 10.1002/mds.28670. Epub 2021 Jun 11."}]}, 'descriptionModule': {'briefSummary': 'The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in participants with spinocerebellar ataxia (SCA).', 'detailedDescription': 'The study was conducted in 2 phases: Randomization Phase (8 weeks) followed by an open-label Extension Phase (336 weeks). During the Randomization Phase, participants received either Troriluzole 140 mg or matching placebo up to 8 weeks. Participants who agreed to enter the Open-label Extension Phase continued dosing of Troriluzole 140 mg for 336 weeks. The study was subsequently amended to follow participants for a total of 336 weeks in the Open-label Extension Phase.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Participants with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10\n* Ability to ambulate 8 meters without assistance (canes and other devices allowed)\n* Screening total Scale for the Assessment and Rating of Ataxia (SARA) score ≥8\n* Score of ≥ 2 on the gait subsection of the SARA\n* Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed\n\nKey Exclusion Criteria:\n\n* Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly explain or contribute significantly to the participants symptoms of ataxia\n* Mini Mental State Exam (MMSE) score \\< 24\n* SARA total score of \\> 30 points at screening\n* Clinical history of stroke\n* Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant"}, 'identificationModule': {'nctId': 'NCT02960893', 'briefTitle': 'Trial in Adult Participants With Spinocerebellar Ataxia (SCA)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Biohaven Pharmaceuticals, Inc.'}, 'officialTitle': 'A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia', 'orgStudyIdInfo': {'id': 'BHV4157-201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Troriluzole - Randomization Phase', 'description': 'Troriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks.', 'interventionNames': ['Drug: Troriluzole']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo - Randomization Phase', 'description': 'Placebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks.', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Troriluzole/Troriluzole - OLE (Open Label Extension) Phase', 'description': 'Participants received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.', 'interventionNames': ['Drug: Troriluzole']}, {'type': 'EXPERIMENTAL', 'label': 'Placebo/Troriluzole - OLE Phase', 'description': 'Participants who received placebo during randomization phase, received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.', 'interventionNames': ['Drug: Placebo', 'Drug: Troriluzole']}], 'interventions': [{'name': 'Troriluzole', 'type': 'DRUG', 'description': 'Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.', 'armGroupLabels': ['Troriluzole - Randomization Phase', 'Troriluzole/Troriluzole - OLE (Open Label Extension) Phase']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.', 'armGroupLabels': ['Placebo - Randomization Phase', 'Placebo/Troriluzole - OLE Phase']}, {'name': 'Troriluzole', 'type': 'DRUG', 'description': 'OLE phase: Neat capsule or formulated capsule (i.e., drug substance with excipients).', 'armGroupLabels': ['Placebo/Troriluzole - OLE Phase', 'Troriluzole/Troriluzole - OLE (Open Label Extension) Phase']}]}, 'contactsLocationsModule': {'locations': [{'zip': '85013', 'city': 'Phoenix', 'state': 'Arizona', 'country': 'United States', 'facility': "St. Joseph's Hospital and Medical Center", 'geoPoint': {'lat': 33.44838, 'lon': -112.07404}}, {'zip': '90806', 'city': 'Long Beach', 'state': 'California', 'country': 'United States', 'facility': 'CNS Trial', 'geoPoint': {'lat': 33.76696, 'lon': -118.18923}}, {'zip': '90095', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Los Angeles', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Francisco', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '80045', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'University of Colorado Denver', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '32611', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}, {'zip': '33612', 'city': 'Tampa', 'state': 'Florida', 'country': 'United States', 'facility': 'University of South Florida', 'geoPoint': {'lat': 27.94752, 'lon': -82.45843}}, {'zip': '30329', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'Northwestern University', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '60637', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'University of Chicago', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins University', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02114', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Harvard University (Massachusetts General Hospital)', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '02215', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Harvard University (Beth Israel Deaconess Medical Center)', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48105', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'University of Michigan', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Columbia University', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '14642', 'city': 'Rochester', 'state': 'New York', 'country': 'United States', 'facility': 'University of Rochester Medical Center', 'geoPoint': {'lat': 43.15478, 'lon': -77.61556}}, {'zip': '75390', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'University of Texas Southwestern', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'Houston Methodist Research Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Biohaven Pharmaceuticals, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}