Troriluzole in Adult Subjects With Spinocerebellar Ataxia

Spinocerebellar Ataxias Clinical Trial

Official title:

A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Subjects With Spinocerebellar Ataxia.

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03701399
• Other study ID #: BHV4157-206
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: March 8, 2019
• Est. completion date: October 2025

Study information

• Verified date: January 2024
• Source: Biohaven Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to compare the efficacy of Troriluzole (200mg once daily) versus placebo after 48 weeks of treatment in subjects with spinocerebellar ataxia (SCA).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 218
• Est. completion date: October 2025
• Est. primary completion date: February 18, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

1. Subjects with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10; currently only enrolling SCA 1, SCA2, SCA3, SCA7, and SCA10 (the cap has been met for SCA6 and SCA8 (on May 31, 2019));

1. A subject should have a confirmed genotypic diagnosis from a CLIA certified lab (can produce test results); or,

2. A subject has a family member that has a confirmed genotypic diagnosis from a CLIA certified lab (can produce test results) and must be willing to undergo genetic testing to confirm underlying SCA diagnosis; or,

3. A subject has a confirmed genotypic diagnosis from a lab that is not CLIA certified and must be willing to undergo genetic testing to confirm underlying SCA diagnosis; or,

4. A subject has clinical evidence that supports diagnosis of one of the aforementioned SCA genotypes but does not have producible test results from a CLIA certified lab from either a family member or for his or herself and the subject must be willing to undergo such testing to confirm the SCA diagnosis (in this case, site must wait for results of genotypic testing prior to randomization)

2. Ability to ambulate 8 meters without human assistance (canes and other devices allowed)

3. Screening f-SARA total score =3;

4. Score of =1 on gait subsection of the f-SARA

5. Determined by the investigator to be medically stable at Baseline/randomization as assessed by medical history, physical examination, laboratory test results, and electrocardiogram testing.

Exclusion Criteria:

1. A = 2-point difference on the Modified Functional SARA score between screening and baseline

2. MMSE score <24

3. Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly explain or contribute significantly to the subjects' symptoms of ataxia.

4. A prominent spasticity or dystonia that, in the opinion of the investigator, will compromise the ability of the SARA instrument to assess underlying ataxia severity.

5. A score of 4 on any individual item (Items 1-4) of the f-SARA

6. 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.

7. Active liver disease or a history of hepatic intolerance to medications that in the investigator's judgment, is medically significant

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia Type 1
• Spinocerebellar Ataxia Type 10
• Spinocerebellar Ataxia Type 2
• Spinocerebellar Ataxia Type 3
• Spinocerebellar Ataxia Type 6
• Spinocerebellar Ataxia Type 7
• Spinocerebellar Ataxia Type 8
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

Drug:
• troriluzole
200 mg by mouth
• Placebos
200 mg by mouth

Locations

Country	Name	City	State
China	Central South University Xiangya Hospital	Changsha	Hunan
China	West China Hospital of Sichuan University	Chengdu	Sichuan
United States	University of Michigan	Ann Arbor	Michigan
United States	Emory	Atlanta	Georgia
United States	University of Colorado Hospital	Aurora	Colorado
United States	Beth Israel Deaconess Medical Center	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	Northwestern University	Chicago	Illinois
United States	University of Chicago	Chicago	Illinois
United States	Duke University Movement Disorders Clinic	Durham	North Carolina
United States	University of Florida Health	Gainesville	Florida
United States	Houston Methodist	Houston	Texas
United States	Mayo Clinic Florida	Jacksonville	Florida
United States	CNS Trials	Long Beach	California
United States	UCLA	Los Angeles	California
United States	Johns Hopkins Medicine	Lutherville	Maryland
United States	Columbia University	New York	New York
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Barrow Neurological Institute	Phoenix	Arizona
United States	Northwest Neurology, Ltd.	Rolling Meadows	Illinois
United States	UCSF	San Francisco	California
United States	Swedish Health Services	Seattle	Washington
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Biohaven Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in the total score of the Modified Functional Scale for the Assessment and Rating of Ataxia (f-SARA) after 48 weeks of treatment.	An increase in the total score indicates a worsening of symptoms.	Baseline to week 48
Secondary	1. Change from baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) score at Randomization Phase Week 48	An increase in the total score indicates a worsening of symptoms.	Baseline to week 48
Secondary	Change from baseline in Activities of Daily Living Scale from the Friedreich's Ataxia Rating Scale (FARS-ADL) at Randomization Week 48.	An increase in the total score indicates a worsening of symptoms.	Baseline to week 48
Secondary	Change from baseline in Functional Staging for Ataxia from the Friedreich's Ataxia Rating Scale (FARS-FUNC) at Randomization Phase Week 48	An increase in the total score indicates a worsening of symptoms.	Baseline to week 48
Secondary	4. Frequency of subjects with the following adverse events (AEs) identified from case report forms: AEs (by severity; by relationship to study drug; overall); SAEs; and AEs leading to treatment discontinuation.		Baseline to week 48