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A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy (VALOR-HCM)

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ClinicalTrials.gov Identifier: NCT04349072
Recruitment Status : Recruiting
First Posted : April 16, 2020
Last Update Posted : June 7, 2021
Sponsor:
Information provided by (Responsible Party):
MyoKardia, Inc.

Tracking Information
First Submitted Date  ICMJE April 7, 2020
First Posted Date  ICMJE April 16, 2020
Last Update Posted Date June 7, 2021
Actual Study Start Date  ICMJE June 26, 2020
Estimated Primary Completion Date September 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 30, 2020)
Septal Reduction Therapy (SRT) Status [ Time Frame: 16 Weeks ]
Number of subjects who decide to proceed with SRT prior to or at Week 16 and the number of subjects who remain guideline eligible for SRT at Week 16.
Original Primary Outcome Measures  ICMJE
 (submitted: April 15, 2020)
Septal Reduction Therapy (SRT) Status [ Time Frame: 16 Weeks ]
The primary endpoint will be a composite of the number of participants that decide to proceed with SRT prior to or at Week 16 and the number of participants that remain guideline eligible for SRT at Week 16.
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: July 7, 2020)
  • SRT Status [ Time Frame: 32 Weeks ]
    Number of subjects who decide to proceed with SRT prior to or at Week 32 and the number of subjects who remain guideline eligible for SRT at Week 32.
  • Mavacamten effect on NYHA [ Time Frame: 16 Weeks ]
    Change from baseline to Week 16 in the mavacamten group compared with the placebo group in New York Heart Association (NYHA) class
  • Mavacamten effect on KCCQ [ Time Frame: 16 Weeks ]
    Change from baseline to Week 16 in the mavacamten group compared with the placebo group in Kansas City Cardiomyopathy Questionnaire (KCCQ-23)
  • Mavacamten effect on NT-proBNP [ Time Frame: 16 Weeks ]
    Change from baseline to Week 16 in the mavacamten group compared with the placebo group in NT-proBNP and cardiac troponin
  • Mavacamten effect on LVOT gradient [ Time Frame: 16 Weeks ]
    Change from baseline to Week 16 in the mavacamten group compared with the placebo group in left ventricular outflow tract (LVOT) gradient
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy
Official Title  ICMJE A Randomized, Double-blind, Placebo-controlled Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Who Are Eligible for Septal Reduction Therapy
Brief Summary This is a randomized, double-blind, placebo-controlled, multi-center study in the United States (U.S.) that will evaluate the effect of mavacamten treatment on reducing the number of septal reduction therapy (SRT) procedures performed in subjects with symptomatic obstructive hypertrophic cardiomyopathy (oHCM [also known as HOCM]) who are eligible for SRT based on ACCF/AHA 2011 and/or ESC 2014 guidelines.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
This is a parallel group treatment study with 2 treatment groups; subjects and investigators are blinded to treatment and dose for the first 16 weeks of treatment. Mavacamten dose is blinded throughout the study.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE HOCM, Hypertrophic Obstructive Cardiomyopathy
Intervention  ICMJE
  • Drug: Mavacamten
    Mavacamten Capsules Other names: MYK-461
  • Drug: Placebo
    Placebo
Study Arms  ICMJE
  • Experimental: Drug: Mavacamten

    Mavacamten Capsules

    Other names:

    MYK-461

    Intervention: Drug: Mavacamten
  • Placebo Comparator: Drug: Placebo
    Matching Placebo Capsules
    Intervention: Drug: Placebo
Publications * Zampieri M, Argirò A, Marchi A, Berteotti M, Targetti M, Fornaro A, Tomberli A, Stefàno P, Marchionni N, Olivotto I. Mavacamten, a Novel Therapeutic Strategy for Obstructive Hypertrophic Cardiomyopathy. Curr Cardiol Rep. 2021 Jun 3;23(7):79. doi: 10.1007/s11886-021-01508-0. Review.

*   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: April 15, 2020)
100
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2024
Estimated Primary Completion Date September 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • At least 18 years old at screening and body weight > 45 kg at screening
  • Diagnosed with oHCM consistent with current ACCF/AHA 2011 and/or ESC 2014 guidelines and meet their recommendations for invasive therapies
  • Referred or under active consideration within the past 12 months for SRT procedure and willing to have SRT procedure
  • Has documented left ventricular ejection fraction (LVEF) ≥ 60% at Screening
  • Has documented oxygen saturation at rest ≥ 90% at Screening

Key Exclusion Criteria:

  • Persistent or permanent atrial fibrillation and subject not on anticoagulation for ≥ 4 weeks prior to screening and/or not adequately rate controlled ≤ 6 months prior to screening
  • Previously treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA])
  • For individuals on beta blockers, calcium channel blockers, or disopyramide, any dose adjustment of these medications < 14 days prior to screening or an anticipated change in regimen during the first 16 weeks of the study
  • Any medical condition that precludes upright exercise stress testing
  • Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at screening
  • Prior treatment with cardiotoxic agents, such as doxorubicin or similar
  • Has a history or evidence of any other clinically significant disorder, condition, or disease that would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: BMS Medical Information Team (Use email contact) clinical.trials@bms.com
Contact: Myokardia Medical Information Team Study Director
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04349072
Other Study ID Numbers  ICMJE MYK-461-017
Has Data Monitoring Committee Yes
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 MyoKardia, Inc.
Study Sponsor  ICMJE MyoKardia, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account MyoKardia, Inc.
Verification Date June 2021

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