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Trial in Adult Subjects With Spinocerebellar Ataxia

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ClinicalTrials.gov Identifier: NCT02960893
Recruitment Status : Active, not recruiting
First Posted : November 10, 2016
Last Update Posted : August 8, 2018
Sponsor:
Collaborators:
Cognitive Research Corporation
Cytel Inc.
Information provided by (Responsible Party):
Biohaven Pharmaceuticals, Inc.

Study Description
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Brief Summary:
The primary purpose of this study is to compare the efficacy of BHV-4157 versus placebo after 8 weeks of treatment on ataxia symptoms in subjects with spinocerebellar ataxia (SCA).

Condition or disease Intervention/treatment Phase
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 Drug: BHV-4157 Drug: Placebo Comparator Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 141 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of BHV-4157 in Adult Subjects With Spinocerebellar Ataxia
Study Start Date : December 2016
Actual Primary Completion Date : August 18, 2017
Estimated Study Completion Date : August 2019

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: BHV-4157
Participants orally receive once daily (QD) dose of BHV-4157 140 milligram (mg) provided as a loose filled capsule in the morning, without regard to meals for 8 weeks. Participants who do not tolerate their treatment switch to night time dosing if there is reason to believe that may help tolerability. In addition, the investigator may permit up to one week of every other day dosing prior to re-instituting daily dosing.
 Drug: BHV-4157
Loose filled capsule
Placebo Comparator: Placebo Comparator
Participants orally receive QD dose of BHV-4157 placebo-matching capsules provided as a loose filled capsule in the morning, without regard to meals for 8 weeks. Participants who do not tolerate their treatment switch to night time dosing if there is reason to believe that may help tolerability. In addition, the investigator may permit up to one week of every other day dosing prior to re-instituting daily dosing.
 Drug: Placebo Comparator
BHV-4157 placebo-matching loose filled capsule


Outcome Measures
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Primary Outcome Measures :
  1. To measure the change in total score on the Scale for Assessment and Rating of Ataxia (SARA) [ Time Frame: The change in total score from baseline to week 8. ]

Secondary Outcome Measures :
  1. • To assess the safety and tolerability of BHV-4157 in subjects with SCA by measuring the frequency and severity of adverse events and discontinuations of adverse events. [ Time Frame: Baseline to week 8. ]
    Measured by the frequency and severity of adverse events and discontinuations of adverse events.

  2. To compare efficacy of BHV-4157 with placebo on patient impression of benefit via use of the PGI-C [ Time Frame: Baseline to Week 8 ]
    Change in PGI-C score


Eligibility Criteria
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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:

  • Subjects with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3 (A Maximum of 12 patients will be enrolled with this genotype- FEB 1 2017 -THIS CAP HAS BEEN MET FOR SCA3), SCA6, SCA7, SCA8 and SCA10
  • Ability to ambulate 8 meters without assistance (canes and other devices allowed)
  • Screening total SARA total score ≥8
  • Determined by the investigator to be medically stable at baseline/randomization and must be physically able and expected to complete the trial as designed
  • Subjects must have adequate hearing, vision, and language skills to perform Scale for the Assessment and Rating of Ataxia (SARA) ratings, 8 Meter Walk Test and other neuropsychiatric testing and interviews as specified in the protocol

Exclusion Criteria:

  • 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
  • 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
Contacts and Locations
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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): NCT02960893


Locations
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United States, Arizona
St. Joseph's Hospital and Medical Center
Phoenix, Arizona, United States, 85013
United States, California
CNS Trial
Long Beach, California, United States, 90806
University of California, Los Angeles
Los Angeles, California, United States, 90095
University of California, San Francisco
San Francisco, California, United States, 94158
United States, Colorado
University of Colorado Denver
Denver, Colorado, United States, 80045
United States, Florida
University of Florida
Gainesville, Florida, United States, 32611
University of South Florida
Tampa, Florida, United States, 33612
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30329
United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
University of Chicago
Chicago, Illinois, United States, 60637
United States, Maryland
Johns Hopkins University
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Harvard University (Massachusetts General Hospital)
Boston, Massachusetts, United States, 02114
Harvard University (Beth Israel Deaconess Medical Center)
Boston, Massachusetts, United States, 02215
United States, Michigan
University of Michigan
Ann Arbor, Michigan, United States, 48105
United States, New York
Columbia University
New York, New York, United States, 10032
University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, Texas
University of Texas Southwestern
Dallas, Texas, United States, 75390
Houston Methodist Research Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Biohaven Pharmaceuticals, Inc.
Cognitive Research Corporation
Cytel Inc.
More Information
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Responsible Party: Biohaven Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02960893     History of Changes
Other Study ID Numbers: BHV4157-201
First Posted: November 10, 2016    Key Record Dates
Last Update Posted: August 8, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
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