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Trial record 8 of 19 for:    biohaven

Troriluzole in Adult Subjects With Spinocerebellar Ataxia

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ClinicalTrials.gov Identifier: NCT03701399
Recruitment Status : Recruiting
First Posted : October 10, 2018
Last Update Posted : July 11, 2019
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 : 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 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 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 increase in 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 BHV-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.



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 (the cap has been met for SCA3 (on May 28, 2019), SCA6, SCA7, SCA8 and SCA10 (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: Robert Berman, MD 203-404-0410 clinicaltrials@biohavenpharma.com/

Locations
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United States, Arizona
Barrow Neurological Institute Recruiting
Phoenix, Arizona, United States, 85013
Contact: Giulia Andrews       Giulia.Andrews@dignityhealth.org   
United States, California
CNS Trials Recruiting
Long Beach, California, United States, 90806
Contact: Anne Cabral    562-304-1742    annecabral@cnstrial.com   
UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Aaron Fisher    310-206-8153    adfisher@mednet.ucla.edu   
UCSF Recruiting
San Francisco, California, United States, 94158
Contact: Nancy Cai    415-502-7640    Nancy.Cai@ucsf.edu   
United States, Colorado
University of Colorado Hospital Recruiting
Aurora, Colorado, United States, 80045
Contact: Nicola Haakonsen    303-724-6351    NICOLA.HAAKONSEN@UCDENVER.EDU   
United States, Florida
University of Florida Health Recruiting
Gainesville, Florida, United States, 32610
Contact: Stephen Gullett    352-273-5550    Stephen.Gullett@neurology.ufl.edu   
Mayo Clinic Florida Recruiting
Jacksonville, Florida, United States, 32224
Contact: Audrey Stongosky, MD    904-953-7229    Strongosky.Audrey2@mayo.edu   
University of South Florida 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: Mildred Valentine    773-702-4610    mvalenti@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 Recruiting
Boston, Massachusetts, United States, 02114
Contact: Jason MacMore    617-726-5060    jmacmore@mgh.harvard.edu   
Beth Israel Deaconess Medical Center Not yet recruiting
Boston, Massachusetts, United States, 02215
Contact: Wilanda Gabriel    617-975-7637    wgabriel@bidmc.harvard.edu   
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Tasha Kaiser       kaisert@med.umich.edu   
United States, New York
Columbia University Recruiting
New York, New York, United States, 10032
Contact: Darya Tomishon    212-305-5779    dvt2101@cumc.columbia.edu   
United States, North Carolina
Duke University Movement Disorders Clinic Not yet recruiting
Durham, North Carolina, United States, 27705
Contact: Burton Scott    919-668-2879    burton.scott@duke.edu   
United States, Pennsylvania
University of Pennsylvania Not yet recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Marcela Pavon    215-829-7725    Marcela.Pavon@uphs.upenn.edu   
United States, Texas
Houston Methodist Recruiting
Houston, Texas, United States, 77030
Contact: Titilayo Olubajo    713-363-9803    tolubajo@houstonmethodist.org   
United States, Washington
Swedish Health Services Recruiting
Seattle, Washington, United States, 98122
Contact: Laura Johnson    206-320-7115    Laura.Johnson3@swedish.org   
Sponsors and Collaborators
Biohaven Pharmaceuticals, Inc.

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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: July 11, 2019
Last Verified: July 2019

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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