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Trial 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. Read our disclaimer for details.
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.

Tracking Information
First Submitted Date  ICMJE November 4, 2016
First Posted Date  ICMJE November 10, 2016
Last Update Posted Date August 8, 2018
Study Start Date  ICMJE December 2016
Actual Primary Completion Date August 18, 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 8, 2016)
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. ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02960893 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 22, 2017)
  • • 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.
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: November 8, 2016)
To measure the total time of the 8 Meter Walk [ Time Frame: The change in total time from baseline to week 8. ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Trial in Adult Subjects With Spinocerebellar Ataxia
Official Title  ICMJE A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of BHV-4157 in Adult Subjects With Spinocerebellar Ataxia
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • 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
Intervention  ICMJE
  • Drug: BHV-4157
    Loose filled capsule
  • Drug: Placebo Comparator
    BHV-4157 placebo-matching loose filled capsule
Study Arms  ICMJE
  • 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.
    Intervention: Drug: BHV-4157
  • 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.
    Intervention: Drug: Placebo Comparator
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: July 30, 2018)
141
Original Estimated Enrollment  ICMJE
 (submitted: November 8, 2016)
120
Estimated Study Completion Date  ICMJE August 2019
Actual Primary Completion Date August 18, 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02960893
Other Study ID Numbers  ICMJE BHV4157-201
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Biohaven Pharmaceuticals, Inc.
Study Sponsor  ICMJE Biohaven Pharmaceuticals, Inc.
Collaborators  ICMJE
  • Cognitive Research Corporation
  • Cytel Inc.
Investigators  ICMJE Not Provided
PRS Account Biohaven Pharmaceuticals, Inc.
Verification Date August 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP