Troriluzole in Adult Subjects With Spinocerebellar Ataxia
A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Subjects With Spinocerebellar Ataxia.
The purpose of this study is to compare the efficacy of Troriluzole (200mg once daily) versus
placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).
Change from Baseline in the total score of the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) after 48 weeks of treatment.
1. Change from baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) score at Randomization Phase Week 48
Study Arms / Comparison Groups
Arm 1: BHV-4157
Description: Troriluzole 200mg PO
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
March 8, 2019
November 30, 2021
Primary Completion Date
November 30, 2021
1. Subjects with a known or suspected diagnosis of the following specific hereditary
ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10; currently only enrolling SCA 1,
SCA2, SCA3, SCA7, and SCA10 (the cap has been met for SCA6 and SCA8 (on May 31,
1. A subject should have a confirmed genotypic diagnosis from a CLIA certified lab
(can produce test results); or,
2. A subject 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 subject 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 subject 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
subject 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
2. Ability to ambulate 8 meters without human assistance (canes and other devices
3. Screening 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
1. A ≥ 2-point difference on the Modified Functional SARA score between screening and
2. MMSE score <24
3. Any medical condition other than one of the hereditary ataxias specified in the
inclusion criteria that could predominantly explain or contribute significantly to the
subjects' symptoms of ataxia.
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. Subjects 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