Troriluzole in Adult Subjects With Spinocerebellar Ataxia

Brief Title

Troriluzole in Adult Subjects With Spinocerebellar Ataxia

Official Title

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

Brief 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).
    


Study Phase

Phase 3

Study Type

Interventional


Primary Outcome

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.

Secondary Outcome

 1. Change from baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) score at Randomization Phase Week 48

Condition

Spinocerebellar Ataxias

Intervention

troriluzole

Study Arms / Comparison Groups

 Arm 1: BHV-4157
Description:  Troriluzole 200mg PO

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

210

Start Date

March 8, 2019

Completion Date

November 30, 2021

Primary Completion Date

November 30, 2021

Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - 75 Years

Accepts Healthy Volunteers

No

Contacts

, 203-404-0410, [email protected]/

Location Countries

China

Location Countries

China

Administrative Informations


NCT ID

NCT03701399

Organization ID

BHV4157-206


Responsible Party

Sponsor

Study Sponsor

Biohaven Pharmaceuticals, Inc.


Study Sponsor

, , 


Verification Date

September 2020