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At Bolder Science, we want your clinical trial search experience to be the best it can be. Complete the following prompts to easily find the trials you are interested in and see trials recruiting near you. You can adjust these selections in your dashboard after creating your account.

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

  • Clinicaltrials.gov identifier

    NCT04349072

  • Recruitment Status

    Active, not recruiting

  • First Posted

    April 16, 2020

  • Last update posted

    October 5, 2021

Study Description

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.

  • Condition or Disease:HOCM, Hypertrophic Obstructive Cardiomyopathy
  • Intervention/Treatment: Drug: Mavacamten
    Drug: Placebo
  • Phase: Phase 3

Detailed Description

N/A

Study Design

  • Study Type: Interventional
  • Estimated Enrollment: 100 participants
  • 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
  • Official Title: A Randomized, Double-blind, Placebo-controlled Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Who Are Eligible for Septal Reduction Therapy
  • Actual Study Start Date: June 2020
  • Estimated Primary Completion Date: September 2024
  • Estimated Study Completion Date: December 2024

Arms and interventions

Arm Intervention/treatment
Experimental: Drug: Mavacamten
Mavacamten Capsules Other names: MYK-461
Drug: Mavacamten
Mavacamten Capsules Other names: MYK-461
Placebo Comparator: Drug: Placebo
Matching Placebo Capsules
Drug: Placebo
Placebo

Outcome Measures

  • Primary Outcome Measures: 1. 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.
  • Secondary Outcome Measures: 1. 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.
  • 2. 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
  • 3. 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)
  • 4. 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
  • 5. 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

Eligibility Criteria

  • Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Contacts

Locations

United States, California
Cedars-Sinai Medical Center
Los Angeles

United States, California
Stanford University Medical Center
Stanford

United States, Connecticut
Yale University School of Medicine - Section of Cardiology
New Haven

United States, Florida
Cleveland Clinic Florida
Weston

United States, Massachusetts
Massachusetts General Hospital
Boston

United States, Massachusetts
Brigham and Women's Hospital
Boston

United States, Michigan
University of Michigan Cardiovascular Center
Ann Arbor

United States, Michigan
University of Michigan Cardiovascular Center
Ann Arbor

United States, Michigan
Spectrum Health Hospital
Grand Rapids

United States, Minnesota
Mayo Clinic
Rochester

United States, Missouri
Washington University School of Medicine
Saint Louis

United States, New York
New York University Langone Medical Center
New York

United States, New York
Westchester Medical Center.
Valhalla

United States, New York
Westchester Medical Center
Valhalla

United States, North Carolina
Duke University School of Medicine
Durham

United States, Ohio
Cleveland Clinic
Cleveland

United States, Oregon
Oregon Health & Science University
Portland

United States, Pennsylvania
University of Pennsylvania - Heart and Vascular Center
Philadelphia

United States, Pennsylvania
Allegheny General Hospital
Pittsburgh

United States, Tennessee
Saint Thomas West Hospital
Nashville

United States, Tennessee
Vanderbilt University
Nashville

United States, Utah
Intermountain Medical Center
Murray

Sponsors and Collaborators

MyoKardia, Inc.

More Information

  • Responsible Party: MyoKardia, Inc.
  • ClinicalTrials.gov Identifier: NCT04349072 History of Changes
  • Other Study ID Numbers: MYK-461-017
  • First Posted: April 16, 2020 Key Record Dates
  • Last Update Posted: October 5, 2021
  • Last Verified: June 2021
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Additional relevant MeSH terms: Hypertrophy
    Cardiomyopathy, Hypertrophic
    Cardiomyopathies