New drug could slow rare brain disease that steals balance

NCT ID NCT03701399

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

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

Summary

This phase 3 trial tests whether the drug troriluzole can slow the progression of spinocerebellar ataxia, a rare genetic disorder that affects coordination and balance. About 300 adults with different types of SCA are randomly assigned to take either troriluzole or a placebo daily for 48 weeks. The main goal is to see if the drug improves scores on a scale that measures walking, standing, sitting, and speech.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: troriluzole
What this could lead to: If successful, troriluzole could become the first approved treatment to slow the progression of spinocerebellar ataxia, helping patients maintain mobility and daily function longer.
What could go wrong: This is a late-stage trial, but previous studies for similar drugs have failed. The benefit may be modest, and side effects like dizziness or nausea are possible.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

299 people

The number who actually took part.

Started

Mar 2019

Expected to finish

Aug 2026

An estimate. End dates often move.

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.

Inclusion Criteria: 1. Participants with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10. 1. A participant should have a confirmed genotypic diagnosis from a Clinical Laboratory Improvement Amendments (CLIA) certified lab (can produce test results); or, 2. A participant 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 participant 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 participant 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 participant 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 randomization) 2. Ability to ambulate 8 meters without human assistance (canes and other devices allowed) 3. Screening Modified Functional Scale for the Assessment and Rating of Ataxia (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 electrocardiogram testing. Exclusion Criteria: 1. A ≥ 2-point difference on the Modified Functional SARA score between screening and baseline 2. Mini Mental State Exam (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 participants' 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. Participants 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.

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Conditions

The condition(s) this trial relates to.

Ataxia autosomal dominant cerebellar ataxia cardiac arrest Machado-Joseph disease spinocerebellar ataxia 7 Spinocerebellar ataxia 8 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 type 1 Spinocerebellar ataxia type 10 Spinocerebellar ataxia type 2 Spinocerebellar ataxia type 3 Spinocerebellar ataxia type 6 Spinocerebellar ataxia type 7 Spinocerebellar ataxia type 8 Spinocerebellar ataxias

Contacts and locations

Locations

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States
Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States
CNS Trials

Long Beach, California, 90806, United States
Central South University Xiangya Hospital

Changsha, Hunan, 410008, China
Columbia University

New York, New York, 10032, United States
Duke University Movement Disorders Clinic

Durham, North Carolina, 27705, United States
Emory

Atlanta, Georgia, 30329, United States
Houston Methodist

Houston, Texas, 77030, United States
Johns Hopkins Medicine

Lutherville, Maryland, 21093, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Mayo Clinic Florida

Jacksonville, Florida, 32224, United States
Northwest Neurology, Ltd.

Rolling Meadows, Illinois, 60008, United States
Northwestern University

Chicago, Illinois, 60611, United States
Swedish Health Services

Seattle, Washington, 98122, United States
UCLA

Los Angeles, California, 90095, United States
UCSF

San Francisco, California, 94158, United States
University of Chicago

Chicago, Illinois, 60637, United States
University of Colorado Hospital

Aurora, Colorado, 80045, United States
University of Florida Health

Gainesville, Florida, 32610, United States
University of Michigan

Ann Arbor, Michigan, 48109, United States
University of Pennsylvania

Philadelphia, Pennsylvania, 19107, United States
University of South Florida

Tampa, Florida, 33612, United States
West China Hospital of Sichuan University

Chengdu, Sichuan, 610041, China

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