A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) (MAVERICK-HCM)

July 14, 2022 updated by: MyoKardia, Inc.

A Randomized, Double-blind, Placebo-controlled, Concentration-guided, Exploratory Study of Mavacameten in Patients With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) and Preserved Left Ventricular Ejection Fraction

This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten doses titrated to achieve 1 of 2 target drug concentrations.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

59

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic Arizona
    • California
      • Los Angeles, California, United States, 90048
        • Cedars-Sinai Medical Center (Smidt Heart Institute)
      • Palo Alto, California, United States, 94305
        • Stanford Hospital and Clinics/Stanford University
      • San Francisco, California, United States, 94143
        • University of California, San Francisco
    • Connecticut
      • New Haven, Connecticut, United States, 06520
        • Yale New Haven Hospital
    • Illinois
      • Evanston, Illinois, United States, 60208
        • Northwestern University
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • St. Vincent Medical Group
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals and Clinics
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • University of Maryland Medical System
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham and Women's Hospital
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Michigan Medicine
      • Detroit, Michigan, United States, 48202
        • Henry Ford Hospital
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University School of Medicine
    • New York
      • Mineola, New York, United States, 11501
        • NYU Winthrop Hospital
      • New York, New York, United States, 10016
        • NYU Langone Medical Center
    • North Carolina
      • Charlotte, North Carolina, United States, 28203
        • Carolinas Medical Center
      • Durham, North Carolina, United States, 27713
        • Duke Cardiology at Southpoint
    • Ohio
      • Cincinnati, Ohio, United States, 45219
        • University of Cincinnati Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Bethlehem, Pennsylvania, United States, 18018
        • St. Luke's Cardiology Associates
      • Hershey, Pennsylvania, United States, 17033-0850
        • Penn State Health Milton S. Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • UPMC Presbyterian
    • Texas
      • Dallas, Texas, United States, 75390-9034
        • The University of Texas Southwestern Medical Center at Dallas
      • Houston, Texas, United States, 77030
        • Houston Methodist Hospital
      • Houston, Texas, United States, 77030
        • Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic
    • Utah
      • Murray, Utah, United States, 84107
        • Intermountain Medical Center
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Medical Center
    • Virginia
      • Charlottesville, Virginia, United States, 22908
        • University of Virginia
      • Richmond, Virginia, United States, 23298
        • Virginia Commonwealth University
    • Washington
      • Seattle, Washington, United States, 98195
        • University of Washington Medical Center
    • Wisconsin
      • Madison, Wisconsin, United States, 53713
        • Unity Point Health Meriter Heart and Vascular Institute

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Diagnosed with nHCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15mm at Screening or ≥ 13mm with a positive family history of HCM.
  • Age 18 and greater, Body weight > 45kg
  • Documented LVEF ≥ 55% at the Screening as determined by echo central lab
  • LVOT gradient < 30 mmHg at rest AND during Valsalva AND post-exercise
  • NYHA functional class II or III
  • Elevated NT-proBNP at rest

Key Exclusion Criteria:

  • History of syncope, sustained ventricular tachyarrhythmia with exercise, obstructive coronary artery disease or myocardial infarction within the past 6 months
  • History of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator (ICD) discharge within 6 months prior to Screening
  • Current treatment with disopyramide or ranolazine (within 14 days prior to Screening)
  • Current or planned treatment during the study with a combination of beta-blockers and calcium channel blockers
  • Has been treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening
  • History of resting or post-exercise LVOT >30 mmHg unless subsequently treated by septal reduction
  • Has QTc Fridericia (QTcF) >480 ms or any other ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II)
  • Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate-controlled within 1 year of Screening
  • History of clinically significant malignant disease within 10 years such as non-metastatic cutaneous squamous cell or basal cell carcinoma
  • History or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator or MyoKardia physician, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Group 1
Active Treatment for participants with base target trough concentration
Drug: mavacamten
MYK-461
Other Names:
  • MYK-461
Experimental: Group 2
Active Treatment for participants with higher target trough concentration
Drug: mavacamten
MYK-461
Other Names:
  • MYK-461
Placebo Comparator: Placebo
Placebo Group
Drug: Placebo
Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)
Time Frame: From first dose to 8 weeks following last dose (Up to 24 weeks)
This is the percentage of participants who experienced at least one treatment emergent adverse event (TEAE)
From first dose to 8 weeks following last dose (Up to 24 weeks)
Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE)
Time Frame: From first dose to 8 weeks following last dose (Up to 24 weeks)
This is the percentage of participants who experienced at least one serious treatment-emergent adverse event (STEAE)
From first dose to 8 weeks following last dose (Up to 24 weeks)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

MyoKardia, Inc.

Investigators

  • Study Director: Myokardia Medical Information Team, MyoKardia, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 30, 2018

Primary Completion (Actual)

January 7, 2020

Study Completion (Actual)

January 7, 2020

Study Registration Dates

First Submitted

February 9, 2018

First Submitted That Met QC Criteria

February 16, 2018

First Posted (Actual)

February 22, 2018

Study Record Updates

Last Update Posted (Actual)

August 9, 2022

Last Update Submitted That Met QC Criteria

July 14, 2022

Last Verified

July 1, 2022

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MYK-461-006

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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