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Troriluzole in Adult Subjects With Spinocerebellar Ataxia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03701399
Recruitment Status : Recruiting
First Posted : October 10, 2018
Last Update Posted : January 18, 2020
Sponsor:
Information provided by (Responsible Party):
Biohaven Pharmaceuticals, Inc.

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).

Condition or disease Intervention/treatment Phase
Spinocerebellar Ataxias Spinocerebellar Ataxia Type 1 Spinocerebellar Ataxia Type 2 Spinocerebellar Ataxia Type 3 Spinocerebellar Ataxia Type 6 Spinocerebellar Ataxia Type 7 Spinocerebellar Ataxia Type 8 Spinocerebellar Ataxia Type 10 Drug: troriluzole Drug: Placebos Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 210 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Subjects With Spinocerebellar Ataxia.
Actual Study Start Date : March 8, 2019
Estimated Primary Completion Date : October 31, 2020
Estimated Study Completion Date : October 31, 2020


Arm Intervention/treatment
Experimental: Arm 1: BHV-4157
Troriluzole 200mg PO
Drug: troriluzole
200 mg PO

Placebo Comparator: Arm 2: Placebo
Placebo 200mg PO
Drug: Placebos
200 mg PO




Primary Outcome Measures :
  1. 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. [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.


Secondary Outcome Measures :
  1. 1. Change from baseline in Patient Impression of Function and Activities of Daily Living Scale (PIFAS) score at Randomization Phase Week 48 [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.

  2. Change from baseline in Activities of Daily Living Scale from the Friedreich's Ataxia Rating Scale (FARS-ADL) at Randomization Week 48. [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.

  3. Change from baseline in Functional Staging for Ataxia from the Friedreich's Ataxia Rating Scale (FARS-FUNC) at Randomization Phase Week 48 [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.

  4. 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. [ Time Frame: Baseline to week 48 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03701399


Contacts
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Contact: Elyse Stock, MD 203-404-0410 clinicaltrials@biohavenpharma.com/

Locations
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Sponsors and Collaborators
Biohaven Pharmaceuticals, Inc.

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Responsible Party: Biohaven Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03701399    
Other Study ID Numbers: BHV4157-206
First Posted: October 10, 2018    Key Record Dates
Last Update Posted: January 18, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Biohaven Pharmaceuticals, Inc.:
Ataxia, SCA, Spinocerebellar Ataxia
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Machado-Joseph Disease
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn