Phase 3
N=141
Trial in Adult Participants With Spinocerebellar Ataxia (SCA)
Spinocerebellar Ataxias · Spinocerebellar Ataxia Genotype Type 1 · Spinocerebellar Ataxia Genotype Type 2 · Spinocerebellar Ataxia Genotype Type 3 · Spinocerebellar Ataxia Genotype Type 6
Bottom Line
View on ClinicalTrials.gov: NCT02960893 ↗Enrolled (actual)
141
Serious AEs
9.2%
Results posted
Aug 2020
Primary outcome: Primary: Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8 — -0.810; -1.059 score on a scale — p=0.519
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Troriluzole (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Biohaven Pharmaceuticals, Inc.
- Primary completion
- Aug 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Total Score on the Scale for the Assessment and Rating of Ataxia (SARA) at Randomization Phase Week 8 |
-0.810; -1.059 | 0.519 |
| SECONDARY Number of Participants With Deaths, Serious Adverse Events (SAEs), Adverse Events (AEs) Leading to Discontinuation, and Treatment-Emergent AEs (TEAEs) During the Randomization Phase |
0; 0; 4; 1; 3; 0 | — |
| SECONDARY Number of Participants With Deaths, SAEs, AEs Leading to Discontinuation and TEAEs During the OLE Phase |
2; 1; 11; 9; 9; 4 | — |
| SECONDARY 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 |
3; 23; 19; 126 | — |
| SECONDARY Number of Participants With Impression of Benefit Via Use of the Patient Global Impression of Change (PGI-C) Index Scale During Randomization Phase |
31; 27; 15; 15; 7; 11 | — |
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).
Eligibility Criteria
Key Inclusion Criteria
- 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
Key Exclusion Criteria
- 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
- Mini Mental State Exam (MMSE) score 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
Data sourced from ClinicalTrials.gov (NCT02960893). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.