Trial of Troriluzole in Adult Subjects with Spinocerebellar Ataxia

  • Study Director: Michael Geschwind, MD, PhD
  • Sponsor: Biohaven Pharmaceuticals
  • Recruiting? No. The study is in follow-up only and not accepting new patients
  • Official Study Title: A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Subjects With Spinocerebellar Ataxia
  • Identifier: NCT02960893
  • Conditions Studied: Spinocerebellar Ataxia (SCA)
  • Intervention: BHV-4157 (Troriluzole) is an investigational drug and will be administered orally at a dose of 140 mg daily
  • Phase: Phase 2/3
  • Duration of Participation: Approximately 2 months followed by a 48-week open-label Extension Phase 

Purpose of the Study

The primary purpose of this study is to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

Study Details

Inclusion Criteria:

  • Age 18–75, inclusive
  • Subjects 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

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 subjects' symptoms of ataxia
  • 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

What to Expect


  • A screening visit to confirm eligibility. Approved subjects will complete a baseline visit and then begin the investigational period.
  • Patients will be given a bottle with the medication, with instructions to take one dose a day.
  • At each visit, subjects will be required to return all study medication bottles dispensed at the last visit.
  • Safety, tolerability and efficacy assessments will be performed at all visits.
  • Functional tests and questionnaires relating to symptoms will be performed at all visits.
  • For each visit, local lab blood draw orders will need to be completed before the next study visit will be given.
  • Subjects will be dosed with either drug or placebo for 48 weeks, at which point they will have the option to continue to an open-label phase of the study and receive the drug if they were on placebo previously. This open-label phase will continue for 48 weeks.
  • A follow-up visit 2 weeks after the last dose of medication. Subjects will come in for a visit to confirm safety and study termination status.

BHV-4157-206 participants cannot participate in other clinical trials of investigational compounds. Participants may still be eligible for BHV-4157-206 if they are taking other prescribed medications.

The Frequency of Visits

Every four weeks.

Materials Needed Before Evaluation

Medical history and confirmed genetic diagnosis, or suspected diagnosis based on family history


All study procedures will be reimbursed by the study. Participants will receive a $75 stipend at each visit for their travel and time.

Contact Information

If you are interested in participating in this trial or have any questions, please contact the study coordinator, Chelsea Chen, at 415.353.8231 or [email protected].

Additional information at UCSF Clinical Trials and the Biohaven Hereditary SCA Study supported by Clara Health.