Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 9 of 12 for:    "Spinocerebellar ataxia 2"

Troriluzole 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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03701399
Recruitment Status : Recruiting
First Posted : October 10, 2018
Last Update Posted : October 9, 2019
Sponsor:
Information provided by (Responsible Party):
Biohaven Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE October 5, 2018
First Posted Date  ICMJE October 10, 2018
Last Update Posted Date October 9, 2019
Actual Study Start Date  ICMJE March 8, 2019
Estimated Primary Completion Date October 31, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 11, 2018)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: October 8, 2018)
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 ]
Change History Complete list of historical versions of study NCT03701399 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: March 7, 2019)
  • 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.
  • 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 ]
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: October 8, 2018)
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Troriluzole in Adult Subjects With Spinocerebellar Ataxia
Official Title  ICMJE A Phase III, Long-Term, Randomized, Double-blind, Placebo-controlled Trial of Troriluzole in Adult Subjects With Spinocerebellar Ataxia.
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE
  • 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
Intervention  ICMJE
  • Drug: troriluzole
    200 mg PO
  • Drug: Placebos
    200 mg PO
Study Arms  ICMJE
  • Experimental: Arm 1: BHV-4157
    Troriluzole 200mg PO
    Intervention: Drug: troriluzole
  • Placebo Comparator: Arm 2: Placebo
    Placebo 200mg PO
    Intervention: Drug: Placebos
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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 8, 2018)
230
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 31, 2020
Estimated Primary Completion Date October 31, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
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: Elyse Stock, MD 203-404-0410 clinicaltrials@biohavenpharma.com/
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03701399
Other Study ID Numbers  ICMJE BHV4157-206
Has Data Monitoring Committee Not Provided
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 Not Provided
Responsible Party Biohaven Pharmaceuticals, Inc.
Study Sponsor  ICMJE Biohaven Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Biohaven Pharmaceuticals, Inc.
Verification Date October 2019

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