Active — Not RecruitingNot Applicable

Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control

United States909 participantsStarted 2019-03-18

Plain-language summary

The purpose of this study is to leverage two sources of real-world data (RWD) to assess the effectiveness of troriluzole after three years of treatment in patients with SCA by comparison to an external control of untreated patients who were followed in a natural history cohort. Real world evidence of effectiveness will be assessed from the RWD sources to examine the treatment effects of toriluzole in SCA out to 3 years. Progression rates of SCA differ by genotype and long-term follow-up is needed to assess for potential efficacy in this rare disease.

Who can participate

Age range

18 Years – 75 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Key Inclusion Criteria for troriluzole-treated participants (BHV4157-206): * Between the ages of 18-75 * Genetic confirmation of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8, and SCA10 * Screening f-SARA total score of ≥3 and score of ≥1 on gait item of the f-SARA. * Ability to ambulate 8 meters without human assistance (canes and other devices were allowed) * Subjects initially randomized to troriluzole Key Inclusion Criteria for participants selected from the natural history studies: * Between ages of 18-75 * CRC-SCA: either a genetic confirmation or a diagnosis of SCA 1, 2, 3, 6, 7, 8, and 10 in themselves or a family member; EUROSCA: genetic confirmation of SCA genotypes 1, 2, 3 and 6. Key Exclusion Criteria for troriluzole-treated participants (BHV4157-206 study): * Screening f-SARA score of 4 on any item of the f-SARA * Any other medical condition that could predominantly explain or contribute significantly to the subjects' symptoms of ataxia or that could confound assessment of ataxia symptoms Key Exclusion Criteria for participants selected from the natural history studies: • Treatment with troriluzole

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Since this study is no longer enrolling new participants, is there any other active trial or expanded access program for troriluzole that I might still be eligible to discuss with you?
2This study is comparing people already taking troriluzole to a natural history control group over three years — what does that mean for what we actually know so far about whether troriluzole slows the progression of spinocerebellar ataxia?
3The main outcome being tracked is a functional walking and movement scale called the f-SARA over three years — based on where I am right now on that kind of scale, how meaningful would that measure be for tracking my own progression?
4Since this is a real-world data study rather than a randomized controlled trial, how confident can we be in the results, and does that affect how you'd weigh troriluzole as a treatment option for me?
5Are there currently any standard-of-care treatments or other clinical trials for my specific type of spinocerebellar ataxia that we should consider alongside or before looking at troriluzole?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change from Baseline in the total score of the modified functional Scale for the Assessment and Rating of Ataxia (f-SARA) at Year 3 in troriluzole-treated subjects is compared to natural history subjects from CRC-SCA

Timeframe: Up to 3 years of treatment

Trial details

NCT IDNCT06529146

SponsorBiohaven Therapeutics Ltd.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2024-09-06

View on ClinicalTrials.gov More Spinocerebellar Ataxias trials