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BHV-4157 in Adult Subjects With Spinocerebellar Ataxia

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ClinicalTrials.gov Identifier: NCT03701399
Recruitment Status : Not yet recruiting
First Posted : October 10, 2018
Last Update Posted : November 2, 2018
Sponsor:
Information provided by (Responsible Party):
Biohaven Pharmaceuticals, Inc.

Brief Summary:
The purpose of this study is to compare the efficacy of BHV-4157 (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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 230 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 BHV-4157 in Adult Subjects With Spinocerebellar Ataxia
Estimated Study Start Date : November 15, 2018
Estimated Primary Completion Date : October 31, 2020
Estimated Study Completion Date : October 31, 2020


Arm Intervention/treatment
Experimental: Arm 1: BHV-4157
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 in the total score of the Modified Scale for the Assessment and Rating of Ataxia (SARA) BHV-4157 versus placebo on ataxia symptoms in subjects with spinocerebellar ataxia (SCA) Type 1 and Type 2, 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. Change of total score as measured by the Modified Scale for the Assessment and Rating of Ataxia of BHV-4157 versus placebo on ataxia symptoms in subjects with spinocerebellar ataxia (SCA) of any genotype after 48 weeks of treatment [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.

  2. To assess of the safety and tolerability of BHV-4157 in subjects with SCA by measuring the frequency and severity of adverse events and discontinuations due to adverse events. [ Time Frame: Baseline to week 48 ]
  3. Measure the change in total score of BHV-4157 versus placebo on patient impression of benefit via use of the Patient Impression of Function and Activities of Daily Living Scale (PIFAS). [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.

  4. Measure the change in the Neuro-QOL Fatigue Scale comparing BHV-4157 versus placebo on daily fatigue and activities. [ Time Frame: Baseline to week 48 ]
    A decreasein the total score indicates a worsening of symptoms.

  5. To measure the change on upper extremity function and activities as measured by the Neuro-QOL Upper Extremity Scale for BHV-4157 versus placebo [ Time Frame: Baseline to week 48 ]
    A decrease in the total score indicates a worsening of symptoms.

  6. To measure the change on lower extremity mobility and activities as measured by the Neuro-QOL Lower extremity mobility scale for cBHV-4157 versus placebo. [ Time Frame: Baseline to week 48 ]
    A decrease in the total score indicates a worsening of symptoms.

  7. To measure the change over time comparing BHV-4157 versus placebo on the clinician impression of global functioning via use of the Clinical Global Impression-Global Improvement Scale (CGI-I) [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.

  8. To measure the change over time comparing BHV-4157 versus placebo on patient impression of global functioning as measured by the Patient Global Impression Scale (PGI) [ Time Frame: Baseline to week 48 ]
    A decrease in the total score indicates a worsening of symptoms.

  9. Compare the change of activities of daily living as measured by the Activities of Daily Living Scale from the Friedreich's Ataxia Rating Scale (FARS-ADL) for BHV-4157 versus placebo [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.

  10. To measure the change on daily functioning using the Functional Staging for Ataxia Scale from the Friedreich's Ataxia Rating Scale (FARS-FUNC) for BHV-4157 versus placebo [ Time Frame: Baseline to week 48 ]
    An increase in the total score indicates a worsening of symptoms.



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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:

a. Subjects with a known or suspected diagnosis of the following specific hereditary ataxias: SCA1, SCA2, SCA3, SCA6, SCA7, SCA8 and SCA10.

i. 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). b. Ability to ambulate 8 meters without human assistance (canes and other devices allowed).

c. Screening total SARA score ≥ 8. d. Determined by the investigator to be medically stable at Baseline/randomization as assessed by medical history, physical examination, laboratory test results, and electrocardiogram testing. Subjects must be physically able and expected to complete the trial as designed.

e. Subjects must have adequate hearing, vision, and language skills to perform SARA ratings and other neuropsychiatric testing and interviews as specified in the protocol.

Exclusion Criteria:

  1. MMSE score <24.
  2. Any medical condition other than one of the hereditary ataxias specified in the inclusion criteria that could predominantly significantly to the subjects' symptoms of ataxia (for example, alcoholism, vitamin deficiencies, multiple sclerosis, vascular disease, tumors, paraneoplastic disease, head injury, idiopathic late onset ataxia, multisystem atrophy) or that can confound assessment of ataxia symptoms (for example, stroke, arthritis).
  3. SARA total score of >30 points at screening.
  4. Clinical history of stroke.
  5. Immunocompromised subjects.
  6. 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
Contact: Robert Berman, MD 203-404-0410 clinicaltrials@biohavenpharma.com/

Locations
United States, Arizona
Barrow Neurological Institute Not yet recruiting
Phoenix, Arizona, United States, 85013
Contact: Giulia Andrews       Giulia.Andrews@dignityhealth.org   
United States, California
CNS Trials Not yet recruiting
Long Beach, California, United States, 90806
Contact: Anne Cabral    562-304-1742    annecabral@cnstrial.com   
UCLA Not yet recruiting
Los Angeles, California, United States, 90095
Contact: Aaron Fisher    310-206-8153    adfisher@mednet.ucla.edu   
UCSF Not yet recruiting
San Francisco, California, United States, 94158
Contact: Nancy Cai    415-502-7640    Nancy.Cai@ucsf.edu   
United States, Colorado
University of Colorado Hospital Not yet recruiting
Aurora, Colorado, United States, 80045
Contact: Andrew Ryberg    303-724-8871    andrew.ryberg@ucdenver.edu   
United States, Florida
University of Florida Health Not yet recruiting
Gainesville, Florida, United States, 32610
Contact: Stephen Gullett    352-273-5550    Stephen.Gullett@neurology.ufl.edu   
Mayo Clinic Florida Not yet recruiting
Jacksonville, Florida, United States, 32224
Contact: Zbigniew Wszolek, MD    904-953-7229    Wszolek.Zbigniew@mayo.edu   
University of South Florida Not yet recruiting
Tampa, Florida, United States, 33612
Contact: Mary Freeman    813-974-5909    mfreema4@health.usf.edu   
United States, Georgia
Emory Not yet recruiting
Atlanta, Georgia, United States, 30329
Contact: Carole Seeley    404-727-8748    Carole.seeley@emory.edu   
United States, Illinois
Northwestern University Not yet recruiting
Chicago, Illinois, United States, 60611
Contact: ZsaZsa Brown    312-503-4121    zsazsa.brown@northwestern.edu   
University of Chicago Not yet recruiting
Chicago, Illinois, United States, 60637
Contact: Jacquelyn Hill    773-702-4610    jhill11@neurology.bsd.uchicago.edu   
United States, Maryland
Johns Hopkins Medicine Not yet recruiting
Lutherville, Maryland, United States, 21093
Contact: Vanessa Johnson    410-616-2816    vjohns23@jhmi.edu   
United States, Massachusetts
Massachusetts General Hospital Not yet recruiting
Boston, Massachusetts, United States, 02114
Contact: Jason MacMore    617-726-5060    jmacmore@mgh.harvard.edu   
United States, Michigan
University of Michigan Not yet recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Tasha Kaiser       kaisert@med.umich.edu   
United States, New York
Columbia University Not yet recruiting
New York, New York, United States, 10032
Contact: Darya Tomishon    212-305-5779    dvt2101@cumc.columbia.edu   
United States, Texas
UT Southwestern Medical Center Not yet recruiting
Dallas, Texas, United States, 75390-9036
Contact: Ashley Gerald    214-648-0212    ashley.gerald@utsouthwestern.edu   
Houston Methodist Not yet recruiting
Houston, Texas, United States, 77030
Contact: Titilayo Olubajo    713-363-9803    tolubajo@houstonmethodist.org   
United States, Washington
Swedish Health Services Not yet recruiting
Seattle, Washington, United States, 98122
Contact: Laura Johnson    206-320-7115    Laura.Johnson3@swedish.org   
Sponsors and Collaborators
Biohaven Pharmaceuticals, Inc.

Responsible Party: Biohaven Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03701399     History of Changes
Other Study ID Numbers: BHV4157-206
First Posted: October 10, 2018    Key Record Dates
Last Update Posted: November 2, 2018
Last Verified: November 2018

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:
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