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

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: NCT04349072
Recruitment Status : Recruiting
First Posted : April 16, 2020
Last Update Posted : August 13, 2020
Sponsor:
Information provided by (Responsible Party):
MyoKardia, Inc.

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 Intervention/treatment Phase
HOCM, Hypertrophic Obstructive Cardiomyopathy Drug: Mavacamten Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
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 26, 2020
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : December 2024


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




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



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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

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): NCT04349072


Contacts
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Contact: Myokardia Medical Information Team 6507410900 medinfo@myokardia.com
Contact: Myokardia Medical Information Team Study Director

Locations
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United States, New York
New York University Langone Medical Center Recruiting
New York, New York, United States, 10016
United States, Ohio
Cleveland Clinic Recruiting
Cleveland, Ohio, United States, 44106
United States, Pennsylvania
University of Pennsylvania - Heart and Vascular Center Recruiting
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
MyoKardia, Inc.
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Responsible Party: MyoKardia, Inc.
ClinicalTrials.gov Identifier: NCT04349072    
Other Study ID Numbers: MYK-461-017
First Posted: April 16, 2020    Key Record Dates
Last Update Posted: August 13, 2020
Last Verified: August 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cardiomyopathies
Cardiomyopathy, Hypertrophic
Hypertrophy
Heart Diseases
Cardiovascular Diseases
Pathological Conditions, Anatomical
Aortic Stenosis, Subvalvular
Aortic Valve Stenosis
Heart Valve Diseases