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Hope for ataxia: experimental drug shows promise in Late-Stage trial

NCT ID NCT02960893

Disease control Sponsor: Biohaven Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a drug called troriluzole in 141 adults with spinocerebellar ataxia, a rare disease that affects balance and coordination. Participants took either the drug or a placebo daily for 8 weeks. The main goal was to see if troriluzole could improve symptoms like walking and hand movements, as measured by a standard ataxia rating scale.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

141 people

The number who actually took part.

Started

Dec 2016

Finished

Sep 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 \< 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

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Conditions

The condition(s) this trial relates to.

autosomal dominant cerebellar ataxia Machado-Joseph disease Machado-Joseph disease type 3 spinocerebellar ataxia 7 spinocerebellar ataxia type 1 spinocerebellar ataxia type 10 spinocerebellar ataxia type 2 spinocerebellar ataxia type 6 spinocerebellar ataxia type 8 Spinocerebellar Ataxias

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Spinocerebellar ataxia genotype type 1 Spinocerebellar ataxia genotype type 10 Spinocerebellar ataxia genotype type 2 Spinocerebellar ataxia genotype type 3 Spinocerebellar ataxia genotype type 6 Spinocerebellar ataxia genotype type 7 Spinocerebellar ataxia genotype type 8 Spinocerebellar ataxias

Contacts and locations

Locations

  • CNS Trial

    Long Beach, California, 90806, United States

  • Columbia University

    New York, New York, 10032, United States

  • Emory University

    Atlanta, Georgia, 30329, United States

  • Harvard University (Beth Israel Deaconess Medical Center)

    Boston, Massachusetts, 02215, United States

  • Harvard University (Massachusetts General Hospital)

    Boston, Massachusetts, 02114, United States

  • Houston Methodist Research Center

    Houston, Texas, 77030, United States

  • Johns Hopkins University

    Baltimore, Maryland, 21287, United States

  • Northwestern University

    Chicago, Illinois, 60611, United States

  • St. Joseph's Hospital and Medical Center

    Phoenix, Arizona, 85013, United States

  • University of California, Los Angeles

    Los Angeles, California, 90095, United States

  • University of California, San Francisco

    San Francisco, California, 94158, United States

  • University of Chicago

    Chicago, Illinois, 60637, United States

  • University of Colorado Denver

    Denver, Colorado, 80045, United States

  • University of Florida

    Gainesville, Florida, 32611, United States

  • University of Michigan

    Ann Arbor, Michigan, 48105, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of South Florida

    Tampa, Florida, 33612, United States

  • University of Texas Southwestern

    Dallas, Texas, 75390, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.