New drug could slow rare brain disease that steals balance
NCT ID NCT03701399
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Barrow Neurological Institute
Phoenix, Arizona, 85013, United States
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Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
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CNS Trials
Long Beach, California, 90806, United States
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Central South University Xiangya Hospital
Changsha, Hunan, 410008, China
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Columbia University
New York, New York, 10032, United States
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Duke University Movement Disorders Clinic
Durham, North Carolina, 27705, United States
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Emory
Atlanta, Georgia, 30329, United States
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Houston Methodist
Houston, Texas, 77030, United States
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Johns Hopkins Medicine
Lutherville, Maryland, 21093, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
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Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
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Northwest Neurology, Ltd.
Rolling Meadows, Illinois, 60008, United States
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Northwestern University
Chicago, Illinois, 60611, United States
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Swedish Health Services
Seattle, Washington, 98122, United States
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UCLA
Los Angeles, California, 90095, United States
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UCSF
San Francisco, California, 94158, United States
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University of Chicago
Chicago, Illinois, 60637, United States
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University of Colorado Hospital
Aurora, Colorado, 80045, United States
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University of Florida Health
Gainesville, Florida, 32610, United States
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University of Michigan
Ann Arbor, Michigan, 48109, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19107, United States
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University of South Florida
Tampa, Florida, 33612, United States
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West China Hospital of Sichuan University
Chengdu, Sichuan, 610041, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can targeted brain zaps ease movement problems in a rare ataxia?
- Can we predict how genetic ataxias progress?
- New hope for SCA patients: expanded access to investigational drug troriluzole
- Brain implant that learns could help rare movement disorder
- Could a drug slow rare brain disease? new study uses Real-World data to find out
- Could stem cells help people with spinocerebellar ataxia? new trial aims to find out