Trial in Adult Participants With Spinocerebellar Ataxia (SCA)

NCT ID: NCT02960893

Last Updated: 2025-10-08

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

141 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-12-15

Study Completion Date

2024-09-20

Brief Summary

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The primary purpose of this study was to compare the efficacy of BHV-4157 (Troriluzole) 140 milligrams (mg) once daily versus placebo after 8 weeks of treatment in participants with spinocerebellar ataxia (SCA).

Detailed Description

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The study was conducted in 2 phases: Randomization Phase (8 weeks) followed by an open-label Extension Phase (336 weeks). During the Randomization Phase, participants received either Troriluzole 140 mg or matching placebo up to 8 weeks. Participants who agreed to enter the Open-label Extension Phase continued dosing of Troriluzole 140 mg for 336 weeks. The study was subsequently amended to follow participants for a total of 336 weeks in the Open-label Extension Phase.

Conditions

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Spinocerebellar Ataxias Spinocerebellar Ataxia Genotype Type 1 Spinocerebellar Ataxia Genotype Type 2 Spinocerebellar Ataxia Genotype Type 3 Spinocerebellar Ataxia Genotype Type 6 Spinocerebellar Ataxia Genotype Type 7 Spinocerebellar Ataxia Genotype Type 8 Spinocerebellar Ataxia Genotype Type 10

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Troriluzole - Randomization Phase

Troriluzole - Randomization Phase: Participants received Troriluzole 140 mg capsules orally once daily (QD) for 8 weeks.

Group Type EXPERIMENTAL

Troriluzole

Intervention Type DRUG

Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.

Placebo - Randomization Phase

Placebo - Randomization Phase: Participants received matching placebo capsules orally QD for 8 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.

Troriluzole/Troriluzole - OLE (Open Label Extension) Phase

Participants received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.

Group Type EXPERIMENTAL

Troriluzole

Intervention Type DRUG

Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.

Troriluzole

Intervention Type DRUG

OLE phase: Neat capsule or formulated capsule (i.e., drug substance with excipients).

Placebo/Troriluzole - OLE Phase

Participants who received placebo during randomization phase, received Troriluzole 140 mg capsules orally QD for 48 weeks in the extension period and were allowed to participate in 288 weeks for expanded extension phase, for a total of 336 weeks of open-label treatment.

Group Type EXPERIMENTAL

Placebo

Intervention Type DRUG

Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.

Troriluzole

Intervention Type DRUG

OLE phase: Neat capsule or formulated capsule (i.e., drug substance with excipients).

Interventions

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Troriluzole

Randomization Phase: Neat (i.e., drug substance without excipients); loose filled capsule.

Intervention Type DRUG

Placebo

Drug: Placebo Randomization Phase: Matching placebo loose filled capsule.

Intervention Type DRUG

Troriluzole

OLE phase: Neat capsule or formulated capsule (i.e., drug substance with excipients).

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Participants with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10
* Ability to ambulate 8 meters without assistance (canes and other devices allowed)
* Screening total Scale for the Assessment and Rating of Ataxia (SARA) score ≥8
* Score of ≥ 2 on the gait subsection of the SARA
* Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed

* Mini Mental State Exam (MMSE) score \< 24
* SARA total score of \> 30 points at screening
* Clinical history of stroke
* Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Biohaven Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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St. Joseph's Hospital and Medical Center

Phoenix, Arizona, United States

Site Status

CNS Trial

Long Beach, California, United States

Site Status

University of California, Los Angeles

Los Angeles, California, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

University of Colorado Denver

Denver, Colorado, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

University of South Florida

Tampa, Florida, United States

Site Status

Emory University

Atlanta, Georgia, United States

Site Status

Northwestern University

Chicago, Illinois, United States

Site Status

University of Chicago

Chicago, Illinois, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

Harvard University (Massachusetts General Hospital)

Boston, Massachusetts, United States

Site Status

Harvard University (Beth Israel Deaconess Medical Center)

Boston, Massachusetts, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

Columbia University

New York, New York, United States

Site Status

University of Rochester Medical Center

Rochester, New York, United States

Site Status

University of Texas Southwestern

Dallas, Texas, United States

Site Status

Houston Methodist Research Center

Houston, Texas, United States

Site Status

Countries

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United States

References

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Schmahmann JD, Pierce S, MacMore J, L'Italien GJ. Development and Validation of a Patient-Reported Outcome Measure of Ataxia. Mov Disord. 2021 Oct;36(10):2367-2377. doi: 10.1002/mds.28670. Epub 2021 Jun 11.

Reference Type DERIVED
PMID: 34115419 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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BHV4157-201

Identifier Type: -

Identifier Source: org_study_id

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