BHV-4157 Trial in Adult Subjects with Spinocerebellar Ataxia

  • Study Director: Michael Geschwind, MD, PhD
  • Sponsor: Biohaven Pharmaceutical Holding Company Ltd.
  • Recruiting? No.
  • Official Study Title: A Phase IIb/III, Randomized, Double-blind, Placebo-controlled Trial of BHV-4157 in Adult Subjects with Spinocerebellar Ataxia
  • ClinicalTrials.gov Identifier: NCT02960893
  • Conditions Studied: spinocerebellar ataxia
  • Intervention: Drug BHV-4157 (140mg) and placebo control. Study drug (BHV-4157 or placebo) is an oral drug taken once daily.
  • Phase: Phase 2b/3
  • Duration of Participation: Up to 64 weeks including Phase 1. Phase 2b/3 is approximately one year.

Purpose of the Study

The purpose of this clinical trial is to compare the efficacy of BHV-4157 versus placebo on ataxia symptoms in subjects with spinocerebellar ataxia (SCA).

Study Details

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 – As of 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 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
  • 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

What to Expect

Types of Testing

Neurological and physical examinations, cognitive testing and neuropsychiatric assessments, surveys, ECGs, blood specimen collection, vital signs

The Frequency of Visits

Phase 2b (BHV-4157 or placebo)

  • One screening visit
  • Baseline visit and two monthly visits
  • Two-weeks post last dose visit (unless participant continues to phase 3)

Phase 3 (BHV-4157 open label)

  • Baseline visit (unless continuing from Phase 2b)
  • Three monthly visits
  • Three 3-month visits
  • Two-weeks post last dose visit

Costs

No costs will be charged for any of the study procedures. Study subjects will receive $50 per visit to assist with travel costs.

Contact Information

We are no longer recruiting participants for this trial. For information about other ataxia studies, please contact the study coordinator, Nancy Cai, at 415.502.7640 or [email protected].