Trial in Adult Participants With Spinocerebellar Ataxia (SCA)
A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Participants With Spinocerebellar Ataxia
1 other identifier
interventional
141
1 country
18
Brief Summary
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Dec 2016
Longer than P75 for phase_2
18 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 4, 2016
CompletedFirst Posted
Study publicly available on registry
November 10, 2016
CompletedStudy Start
First participant enrolled
December 15, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 18, 2017
CompletedResults Posted
Study results publicly available
August 14, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
September 20, 2024
CompletedOctober 8, 2025
September 1, 2025
8 months
November 4, 2016
July 24, 2020
September 19, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8
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.
Baseline, Randomization Phase Week 8
Secondary Outcomes (4)
Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase
From first dose of study drug to 30 days post the last dose in the Randomization Phase (Up to 12 weeks)
Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase
From first dose of study drug to 30 days post the last dose in the OLE Phase (Up to 340 weeks)
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
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)
Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase
Randomization Phase Week 8
Other Outcomes (1)
Change From Randomization Baseline in Total Score on the SARA at Extension Phase Week 48
Randomization Baseline, Extension Phase Week 48
Study Arms (4)
Troriluzole - Randomization Phase
EXPERIMENTALTroriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks.
Placebo - Randomization Phase
PLACEBO COMPARATORPlacebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks.
Troriluzole/Troriluzole - OLE (Open Label Extension) Phase
EXPERIMENTALParticipants 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.
Placebo/Troriluzole - OLE Phase
EXPERIMENTALParticipants 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.
Interventions
Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.
Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.
Eligibility Criteria
You may qualify if:
- Participants with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10
- Ability to ambulate 8 meters without assistance (canes and other devices allowed)
- Screening total Scale for the Assessment and Rating of Ataxia (SARA) score ≥8
- Score of ≥ 2 on the gait subsection of the SARA
- Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed
You may not qualify if:
- Mini Mental State Exam (MMSE) score \< 24
- SARA total score of \> 30 points at screening
- Clinical history of stroke
- Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (18)
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, 85013, United States
CNS Trial
Long Beach, California, 90806, United States
University of California, Los Angeles
Los Angeles, California, 90095, United States
University of California, San Francisco
San Francisco, California, 94158, United States
University of Colorado Denver
Denver, Colorado, 80045, United States
University of Florida
Gainesville, Florida, 32611, United States
University of South Florida
Tampa, Florida, 33612, United States
Emory University
Atlanta, Georgia, 30329, United States
Northwestern University
Chicago, Illinois, 60611, United States
University of Chicago
Chicago, Illinois, 60637, United States
Johns Hopkins University
Baltimore, Maryland, 21287, United States
Harvard University (Massachusetts General Hospital)
Boston, Massachusetts, 02114, United States
Harvard University (Beth Israel Deaconess Medical Center)
Boston, Massachusetts, 02215, United States
University of Michigan
Ann Arbor, Michigan, 48105, United States
Columbia University
New York, New York, 10032, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
University of Texas Southwestern
Dallas, Texas, 75390, United States
Houston Methodist Research Center
Houston, Texas, 77030, United States
Related Publications (1)
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.
PMID: 34115419DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Chief Medical Officer
- Organization
- Biohaven Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- LTE60
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
November 4, 2016
First Posted
November 10, 2016
Study Start
December 15, 2016
Primary Completion
August 18, 2017
Study Completion
September 20, 2024
Last Updated
October 8, 2025
Results First Posted
August 14, 2020
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share