Trial in Adult Participants With Spinocerebellar Ataxia (SCA)

Last updated: September 19, 2025
Sponsor: Biohaven Pharmaceuticals, Inc.
Overall Status: Completed

Phase

2/3

Condition

Spinocerebellar Disorders

Friedreich's Ataxia

Dyskinesias

Treatment

Placebo

Troriluzole

Clinical Study ID

NCT02960893
BHV4157-201
  • Ages 18-75
  • All Genders

Study 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

Inclusion

Key Inclusion Criteria:

  • Participants with a known or suspected diagnosis of the following specifichereditary 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 andmust be physically able and expected to complete the trial as designed

Exclusion

Key Exclusion Criteria:

  • Any medical condition other than one of the hereditary ataxias specified in theinclusion criteria that could predominantly explain or contribute significantly tothe participants 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 theinvestigator's judgment, is medically significant

Study Design

Total Participants: 141
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2/3
Study Start date:
December 15, 2016
Estimated Completion Date:
September 20, 2024

Study Description

The study was conducted in 2 phases: Randomization Phase (8 weeks) followed by an open-label Extension Phase (336 weeks). During the Randomization Phase, participants received either Troriluzole 140 mg or matching placebo up to 8 weeks. Participants who agreed to enter the Open-label Extension Phase continued dosing of Troriluzole 140 mg for 336 weeks. The study was subsequently amended to follow participants for a total of 336 weeks in the Open-label Extension Phase.

Connect with a study center

  • St. Joseph's Hospital and Medical Center

    Phoenix, Arizona 85013
    United States

    Site Not Available

  • St. Joseph's Hospital and Medical Center

    Phoenix 5308655, Arizona 5551752 85013
    United States

    Site Not Available

  • CNS Trial

    Long Beach, California 90806
    United States

    Site Not Available

  • University of California, Los Angeles

    Los Angeles, California 90095
    United States

    Site Not Available

  • University of California, San Francisco

    San Francisco, California 94158
    United States

    Site Not Available

  • CNS Trial

    Long Beach 5367929, California 5332921 90806
    United States

    Site Not Available

  • University of California, Los Angeles

    Los Angeles 5368361, California 5332921 90095
    United States

    Site Not Available

  • University of California, San Francisco

    San Francisco 5391959, California 5332921 94158
    United States

    Site Not Available

  • University of Colorado Denver

    Denver, Colorado 80045
    United States

    Site Not Available

  • University of Colorado Denver

    Denver 5419384, Colorado 5417618 80045
    United States

    Site Not Available

  • University of Florida

    Gainesville, Florida 32611
    United States

    Site Not Available

  • University of South Florida

    Tampa, Florida 33612
    United States

    Site Not Available

  • University of Florida

    Gainesville 4156404, Florida 4155751 32611
    United States

    Site Not Available

  • University of South Florida

    Tampa 4174757, Florida 4155751 33612
    United States

    Site Not Available

  • Emory University

    Atlanta, Georgia 30329
    United States

    Site Not Available

  • Emory University

    Atlanta 4180439, Georgia 4197000 30329
    United States

    Site Not Available

  • Northwestern University

    Chicago, Illinois 60611
    United States

    Site Not Available

  • University of Chicago

    Chicago, Illinois 60637
    United States

    Site Not Available

  • Northwestern University

    Chicago 4887398, Illinois 4896861 60611
    United States

    Site Not Available

  • University of Chicago

    Chicago 4887398, Illinois 4896861 60637
    United States

    Site Not Available

  • Johns Hopkins University

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • Johns Hopkins University

    Baltimore 4347778, Maryland 4361885 21287
    United States

    Site Not Available

  • Harvard University (Beth Israel Deaconess Medical Center)

    Boston, Massachusetts 02215
    United States

    Site Not Available

  • Harvard University (Massachusetts General Hospital)

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • Harvard University (Beth Israel Deaconess Medical Center)

    Boston 4930956, Massachusetts 6254926 02215
    United States

    Site Not Available

  • Harvard University (Massachusetts General Hospital)

    Boston 4930956, Massachusetts 6254926 02114
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48105
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor 4984247, Michigan 5001836 48105
    United States

    Site Not Available

  • Columbia University

    New York, New York 10032
    United States

    Site Not Available

  • University of Rochester Medical Center

    Rochester, New York 14642
    United States

    Site Not Available

  • Columbia University

    New York 5128581, New York 5128638 10032
    United States

    Site Not Available

  • University of Rochester Medical Center

    Rochester 5134086, New York 5128638 14642
    United States

    Site Not Available

  • University of Texas Southwestern

    Dallas, Texas 75390
    United States

    Site Not Available

  • Houston Methodist Research Center

    Houston, Texas 77030
    United States

    Site Not Available

  • University of Texas Southwestern

    Dallas 4684888, Texas 4736286 75390
    United States

    Site Not Available

  • Houston Methodist Research Center

    Houston 4699066, Texas 4736286 77030
    United States

    Site Not Available

Map preview placeholder

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.