Troriluzole in Adult Participants With Spinocerebellar Ataxia
NCT ID: NCT03701399
Last Updated: 2025-11-04
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE3
299 participants
INTERVENTIONAL
2019-03-08
2026-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Troriluzole
Randomization phase (Randomization through Week 48):
Participants received starting dose of troriluzole 140 milligrams (mg) capsules orally once daily for first 4 weeks, followed by 200 mg once daily for the remaining 44 weeks of 48-week duration of the double-blind randomization phase.
Open-label extension (OLE) phase (OLE Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 200 mg capsules orally once daily for 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
troriluzole
Administered orally
Placebo
Randomization phase (Randomization through Week 48):
Participants received troriluzole matching placebo capsules orally once daily for 48 weeks duration of the double-blind randomization phase.
OLE phase (Week 1 up to Week 192): Participants who were eligible and agreed to enroll in an OLE phase, will receive troriluzole 140 mg capsules orally once daily for the first 4 weeks in a blinded manner. After OLE Week 4, all participants will then receive open label troriluzole 200 mg up to Week 192.
troriluzole
Administered orally
Placebo
Administered orally
Interventions
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troriluzole
Administered orally
Placebo
Administered orally
Eligibility Criteria
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Inclusion Criteria
1. A participant should have a confirmed genotypic diagnosis from a Clinical Laboratory Improvement Amendments (CLIA) certified lab (can produce test results); or,
2. A participant has a family member that has a confirmed genotypic diagnosis from a CLIA certified lab (can produce test results) and must be willing to undergo genetic testing to confirm underlying SCA diagnosis; or,
3. A participant has a confirmed genotypic diagnosis from a lab that is not CLIA certified and must be willing to undergo genetic testing to confirm underlying SCA diagnosis; or,
4. A participant has clinical evidence that supports diagnosis of one of the aforementioned SCA genotypes but does not have producible test results from a CLIA certified lab from either a family member or for his or herself and the participant must be willing to undergo such testing to confirm the SCA diagnosis (in this case, site must wait for results of genotypic testing prior to randomization)
2. Ability to ambulate 8 meters without human assistance (canes and other devices allowed)
3. Screening Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) total score ≥3.
4. Score of ≥1 on gait subsection of the f-SARA
5. Determined by the investigator to be medically stable at Baseline/randomization as assessed by medical history, physical examination, laboratory test results, and electrocardiogram testing.
4. A prominent spasticity or dystonia that, in the opinion of the investigator, will compromise the ability of the SARA instrument to assess underlying ataxia severity.
5. A score of 4 on any individual item (Items 1-4) of the f-SARA
6. Participants should be excluded at screening or baseline if medical conditions have arisen or there is a change in disease status that could confound the ability of the SARA to accurately reflect changes in ataxia severity.
7. Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant.
Exclusion Criteria
2. Mini Mental State Exam (MMSE) score \<24
18 Years
75 Years
ALL
No
Sponsors
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Biohaven Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Barrow Neurological Institute
Phoenix, Arizona, United States
CNS Trials
Long Beach, California, United States
UCLA
Los Angeles, California, United States
UCSF
San Francisco, California, United States
University of Colorado Hospital
Aurora, Colorado, United States
University of Florida Health
Gainesville, Florida, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
University of South Florida
Tampa, Florida, United States
Emory
Atlanta, Georgia, United States
Northwestern University
Chicago, Illinois, United States
University of Chicago
Chicago, Illinois, United States
Northwest Neurology, Ltd.
Rolling Meadows, Illinois, United States
Johns Hopkins Medicine
Lutherville, Maryland, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Columbia University
New York, New York, United States
Duke University Movement Disorders Clinic
Durham, North Carolina, United States
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Houston Methodist
Houston, Texas, United States
Swedish Health Services
Seattle, Washington, United States
Central South University Xiangya Hospital
Changsha, Hunan, China
West China Hospital of Sichuan University
Chengdu, Sichuan, China
Countries
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References
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Potashman MH, Popoff E, Powell LC, Beiner MW, Mackenzie A, Coric V, Subramony S, Synofzik M, Schmahmann J, L'Italien G. Measurement Properties of the Friedreich Ataxia Rating Scale in Patients with Spinocerebellar Ataxia. Neurol Ther. 2025 Apr;14(2):527-545. doi: 10.1007/s40120-024-00708-4. Epub 2025 Jan 13.
L'Italien GJ, Oikonomou EK, Khera R, Potashman MH, Beiner MW, Maclaine GDH, Schmahmann JD, Perlman S, Coric V. Video-Based Kinematic Analysis of Movement Quality in a Phase 3 Clinical Trial of Troriluzole in Adults with Spinocerebellar Ataxia: A Post Hoc Analysis. Neurol Ther. 2024 Aug;13(4):1287-1301. doi: 10.1007/s40120-024-00625-6. Epub 2024 May 30.
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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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BHV4157-206
Identifier Type: -
Identifier Source: org_study_id
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