- Study Director: Michael Geschwind, MD, PhD
- Sponsor: Biohaven Pharmaceuticals
- Recruiting? Active trial, not recruiting
- Official Study Title: A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of BHV-4157 in Adult Subjects with Spinocerebellar Ataxia
- ClinicalTrials.gov Identifier: NCT03701399
- Conditions Studied: Spinocerebellar Ataxia (SCA)
- Intervention: BHV-4157 (Troriluzole) is an investigational drug and will be administered orally at a dose of 200mg daily
- Phase: Phase 3
- Duration of Participation: Approximately 15 months
Purpose of the Study
The BHV-4157-206 trial is designed to assess safety, tolerability and efficacy signals of BHV-4157 (Troriluzole) in a population of patients with spinocerebellar ataxia (SCA); specifically, SCA1, SCA2, SCA3, SCA7 and SCA10. It is a Phase III, multicenter, randomized, double-blind, 2-arm placebo-controlled parallel-group study at multiple sites across the United States and China. BHV-4157 (Troriluzole) is an adaptation of Riluzole, an FDA-approved medication for amyotrophic lateral sclerosis (ALS).
Study Details
Inclusion Criteria:
- Age 18–75, inclusive
- A positive genetic diagnosis of SCA1, SCA2, SCA3, SCA7 or SCA10
Exclusion Criteria:
- A ≥2-point difference on the Modified Functional SARA score between screening and baseline
- MMSE score <24
- Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could explain or contribute significantly to the subjects’ symptoms of ataxia
- Prominent spasticity or dystonia that, in the opinion of the investigator, will compromise the ability of the SARA instrument to assess underlying ataxia severity
- A score of 4 on any individual item (Items 1–4) of the f-SARA
- Subjects 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
- Active liver disease or a history of hepatic intolerance to medications that in the investigator’s judgment, is medically significant
What to Expect
Testing
- A screening visit to confirm eligibility. Approved subjects will complete a baseline visit and then begin the investigational period.
- Patients will be given a bottle with the medication, with instructions to take one dose a day.
- At each visit, subjects will be required to return all study medication bottles dispensed at the last visit.
- Safety, tolerability and efficacy assessments will be performed at all visits.
- Functional tests and questionnaires relating to symptoms will be performed at all visits.
- On-site blood draws will be performed at all visits.
- Subjects will be dosed with either drug or placebo for 48 weeks, at which point they will have the option to continue to an open-label phase of the study and receive the drug if they were on placebo previously. This open-label phase will continue for 48 weeks.
- A follow-up visit 2 weeks after the last dose of medication. Subjects will come in for a visit to confirm safety and study termination status.
BHV-4157-206 participants cannot participate in other clinical trials of investigational compounds. Participants may still be eligible for BHV-4157-206 if they are taking other prescribed medications.
The Frequency of Visits
Every four weeks
Materials Needed Before Evaluation
Medical history and confirmed genetic diagnosis, or suspected diagnosis based on family history
Costs
All study procedures will be reimbursed by the study. Participants will receive a $75 stipend at each visit for their travel and time.
Contact Information
If you are interested in participating in this trial or have any questions, please contact the study coordinator, Chelsea Chen, at 415.353.8231 or [email protected].
Additional information at UCSF Clinical Trials and the Neighborhoodtrials.com website.
