New hope for SCA patients: expanded access to investigational drug troriluzole

NCT ID NCT06034886

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This expanded access program offers the investigational drug troriluzole to people with spinocerebellar ataxia (SCA), a rare brain disorder that affects movement and coordination. Participants take a daily pill. The goal is to provide access to the drug while it is still being studied. Eligible patients must have a confirmed SCA diagnosis and adequate liver function.

What this could mean

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

Active substance
troriluzole (also known as BHV-4157), taken as a daily pill
What this could lead to
If it works, this could help slow or manage symptoms of spinocerebellar ataxia, a rare and progressive neurological disease.
What could go wrong
This is an expanded access program, not a formal trial, so evidence is limited. The drug is still investigational and may not work for everyone. Liver function must be monitored.

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.

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