Troriluzole in Adult Participants With Spinocerebellar Ataxia

Last updated: January 16, 2025
Sponsor: Biohaven Pharmaceuticals, Inc.
Overall Status: Active - Not Recruiting

Phase

3

Condition

Spinocerebellar Disorders

Friedreich's Ataxia

Dyskinesias

Treatment

Placebo

troriluzole

Placebos

Clinical Study ID

NCT03701399
BHV4157-206
  • Ages 18-75
  • All Genders

Study Summary

The purpose of this study is to compare the efficacy of Troriluzole (200 mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Participants with a known or suspected diagnosis of the following specifichereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10.

  2. A participant should have a confirmed genotypic diagnosis from a ClinicalLaboratory Improvement Amendments (CLIA) certified lab (can produce testresults); or,

  3. A participant has a family member that has a confirmed genotypic diagnosis froma CLIA certified lab (can produce test results) and must be willing to undergogenetic testing to confirm underlying SCA diagnosis; or,

  4. A participant has a confirmed genotypic diagnosis from a lab that is not CLIAcertified and must be willing to undergo genetic testing to confirm underlyingSCA diagnosis; or,

  5. A participant has clinical evidence that supports diagnosis of one of theaforementioned SCA genotypes but does not have producible test results from aCLIA certified lab from either a family member or for his or herself and theparticipant must be willing to undergo such testing to confirm the SCAdiagnosis (in this case, site must wait for results of genotypic testing priorto randomization)

  6. Ability to ambulate 8 meters without human assistance (canes and other devicesallowed)

  7. Screening Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA)total score ≥3.

  8. Score of ≥1 on gait subsection of the f-SARA

  9. Determined by the investigator to be medically stable at Baseline/randomization asassessed by medical history, physical examination, laboratory test results, andelectrocardiogram testing.

Exclusion

Exclusion Criteria:

  1. A ≥ 2-point difference on the Modified Functional SARA score between screening andbaseline

  2. Mini Mental State Exam (MMSE) score <24

  3. 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.

  4. A prominent spasticity or dystonia that, in the opinion of the investigator, willcompromise 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. Participants should be excluded at screening or baseline if medical conditions havearisen or there is a change in disease status that could confound the ability of theSARA to accurately reflect changes in ataxia severity.

  7. Active liver disease or a history of hepatic intolerance to medications that in theinvestigator's judgment, is medically significant.

Study Design

Total Participants: 299
Treatment Group(s): 3
Primary Treatment: Placebo
Phase: 3
Study Start date:
March 08, 2019
Estimated Completion Date:
October 31, 2025

Connect with a study center

  • Central South University Xiangya Hospital

    Changsha, Hunan 410008
    China

    Site Not Available

  • West China Hospital of Sichuan University

    Chengdu, Sichuan 610041
    China

    Site Not Available

  • Barrow Neurological Institute

    Phoenix, Arizona 85013
    United States

    Site Not Available

  • CNS Trials

    Long Beach, California 90806
    United States

    Site Not Available

  • UCLA

    Los Angeles, California 90095
    United States

    Site Not Available

  • UCSF

    San Francisco, California 94158
    United States

    Site Not Available

  • University of Colorado Hospital

    Aurora, Colorado 80045
    United States

    Site Not Available

  • University of Florida Health

    Gainesville, Florida 32610
    United States

    Site Not Available

  • Mayo Clinic Florida

    Jacksonville, Florida 32224
    United States

    Site Not Available

  • University of South Florida

    Tampa, Florida 33612
    United States

    Site Not Available

  • Emory

    Atlanta, Georgia 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

  • Northwest Neurology, Ltd.

    Lake Barrington, Illinois 60010
    United States

    Site Not Available

  • Northwest Neurology, Ltd.

    Rolling Meadows, Illinois 60008
    United States

    Site Not Available

  • Johns Hopkins Medicine

    Lutherville, Maryland 21093
    United States

    Site Not Available

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts 02215
    United States

    Site Not Available

  • Massachusetts General Hospital

    Boston, Massachusetts 02114
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • Columbia University

    New York, New York 10032
    United States

    Site Not Available

  • Duke University Movement Disorders Clinic

    Durham, North Carolina 27705
    United States

    Site Not Available

  • University of Pennsylvania

    Philadelphia, Pennsylvania 19107
    United States

    Site Not Available

  • UT Southwestern Medical Center

    Dallas, Texas 75390-9036
    United States

    Site Not Available

  • Houston Methodist

    Houston, Texas 77030
    United States

    Site Not Available

  • Swedish Health Services

    Seattle, Washington 98122
    United States

    Site Not Available

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