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A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) (MAVERICK-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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03442764
Recruitment Status : Completed
First Posted : February 22, 2018
Results First Posted : August 9, 2022
Last Update Posted : August 9, 2022
Sponsor:
Information provided by (Responsible Party):
MyoKardia, Inc.

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Non-obstructive Hypertrophic Cardiomyopathy Drug: mavacamten Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 59 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : March 30, 2018
Actual Primary Completion Date : January 7, 2020
Actual Study Completion Date : January 7, 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cardiomyopathy

Arms and Interventions
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Arm Intervention/treatment
Experimental: Group 1
Active Treatment for participants with base target trough concentration
 Drug: mavacamten
MYK-461
Other Name: MYK-461
Experimental: Group 2
Active Treatment for participants with higher target trough concentration
 Drug: mavacamten
MYK-461
Other Name: MYK-461
Placebo Comparator: Placebo
Placebo Group
 Drug: Placebo
Placebo


Outcome Measures
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Primary Outcome Measures :
  1. 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)

  2. 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)


Eligibility Criteria
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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:

  • 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.
Contacts and Locations
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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): NCT03442764


Locations
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Sponsors and Collaborators
MyoKardia, Inc.
Investigators
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Study Director: Myokardia Medical Information Team MyoKardia, Inc.
  Study Documents (Full-Text)

Documents provided by MyoKardia, Inc.:
Study Protocol  [PDF] April 4, 2018
Statistical Analysis Plan  [PDF] August 28, 2019

More Information
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Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: MyoKardia, Inc.
ClinicalTrials.gov Identifier: NCT03442764    
Other Study ID Numbers: MYK-461-006
First Posted: February 22, 2018    Key Record Dates
Results First Posted: August 9, 2022
Last Update Posted: August 9, 2022
Last Verified: July 2022

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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
Aortic Valve Disease
Heart Valve Diseases