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

NCT ID: NCT03442764

Last Updated: 2022-08-09

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

59 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-03-30

Study Completion Date

2020-01-07

Brief Summary

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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.

Detailed Description

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Conditions

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Non-obstructive Hypertrophic Cardiomyopathy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Group 1

Active Treatment for participants with base target trough concentration

Group Type EXPERIMENTAL

mavacamten

Intervention Type DRUG

MYK-461

Group 2

Active Treatment for participants with higher target trough concentration

Group Type EXPERIMENTAL

mavacamten

Intervention Type DRUG

MYK-461

Placebo

Placebo Group

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo

Interventions

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mavacamten

MYK-461

Intervention Type DRUG

Placebo

Placebo

Intervention Type DRUG

Other Intervention Names

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MYK-461

Eligibility Criteria

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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

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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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MyoKardia, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Myokardia Medical Information Team

Role: STUDY_DIRECTOR

MyoKardia, Inc.

Locations

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Mayo Clinic Arizona

Scottsdale, Arizona, United States

Site Status

Cedars-Sinai Medical Center (Smidt Heart Institute)

Los Angeles, California, United States

Site Status

Stanford Hospital and Clinics/Stanford University

Palo Alto, California, United States

Site Status

University of California, San Francisco

San Francisco, California, United States

Site Status

Yale New Haven Hospital

New Haven, Connecticut, United States

Site Status

Northwestern University

Evanston, Illinois, United States

Site Status

St. Vincent Medical Group

Indianapolis, Indiana, United States

Site Status

University of Iowa Hospitals and clinics

Iowa City, Iowa, United States

Site Status

University of Maryland Medical System

Baltimore, Maryland, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Michigan Medicine

Ann Arbor, Michigan, United States

Site Status

Henry Ford Hospital

Detroit, Michigan, United States

Site Status

Washington University School of Medicine

St Louis, Missouri, United States

Site Status

NYU Winthrop Hospital

Mineola, New York, United States

Site Status

NYU Langone Medical Center

New York, New York, United States

Site Status

Carolinas Medical Center

Charlotte, North Carolina, United States

Site Status

Duke Cardiology at Southpoint

Durham, North Carolina, United States

Site Status

University of Cincinnati Medical Center

Cincinnati, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

St. Luke's Cardiology Associates

Bethlehem, Pennsylvania, United States

Site Status

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

UPMC Presbyterian

Pittsburgh, Pennsylvania, United States

Site Status

The University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Site Status

Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic

Houston, Texas, United States

Site Status

Houston Methodist Hospital

Houston, Texas, United States

Site Status

Intermountain Medical Center

Murray, Utah, United States

Site Status

University of Utah Medical Center

Salt Lake City, Utah, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

University of Washington Medical Center

Seattle, Washington, United States

Site Status

Unity Point Health Meriter Heart and Vascular Institute

Madison, Wisconsin, United States

Site Status

Countries

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United States

References

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Ho CY, Mealiffe ME, Bach RG, Bhattacharya M, Choudhury L, Edelberg JM, Hegde SM, Jacoby D, Lakdawala NK, Lester SJ, Ma Y, Marian AJ, Nagueh SF, Owens A, Rader F, Saberi S, Sehnert AJ, Sherrid MV, Solomon SD, Wang A, Wever-Pinzon O, Wong TC, Heitner SB. Evaluation of Mavacamten in Symptomatic Patients With Nonobstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2020 Jun 2;75(21):2649-2660. doi: 10.1016/j.jacc.2020.03.064.

Reference Type DERIVED
PMID: 32466879 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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MYK-461-006

Identifier Type: -

Identifier Source: org_study_id

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