A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)
MAVERICK-HCM
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
1 other identifier
interventional
59
1 country
32
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Mar 2018
32 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 9, 2018
CompletedFirst Posted
Study publicly available on registry
February 22, 2018
CompletedStudy Start
First participant enrolled
March 30, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 7, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
January 7, 2020
CompletedResults Posted
Study results publicly available
August 9, 2022
CompletedAugust 9, 2022
July 1, 2022
1.8 years
February 9, 2018
May 27, 2022
July 14, 2022
Conditions
Outcome Measures
Primary Outcomes (2)
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)
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)
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)
Study Arms (3)
Group 1
EXPERIMENTALActive Treatment for participants with base target trough concentration
Group 2
EXPERIMENTALActive Treatment for participants with higher target trough concentration
Placebo
PLACEBO COMPARATORPlacebo Group
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- MyoKardia, Inc.lead
Study Sites (32)
Mayo Clinic Arizona
Scottsdale, Arizona, 85259, United States
Cedars-Sinai Medical Center (Smidt Heart Institute)
Los Angeles, California, 90048, United States
Stanford Hospital and Clinics/Stanford University
Palo Alto, California, 94305, United States
University of California, San Francisco
San Francisco, California, 94143, United States
Yale New Haven Hospital
New Haven, Connecticut, 06520, United States
Northwestern University
Evanston, Illinois, 60208, United States
St. Vincent Medical Group
Indianapolis, Indiana, 46260, United States
University of Iowa Hospitals and clinics
Iowa City, Iowa, 52242, United States
University of Maryland Medical System
Baltimore, Maryland, 21201, United States
Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
Michigan Medicine
Ann Arbor, Michigan, 48109, United States
Henry Ford Hospital
Detroit, Michigan, 48202, United States
Washington University School of Medicine
St Louis, Missouri, 63110, United States
NYU Winthrop Hospital
Mineola, New York, 11501, United States
NYU Langone Medical Center
New York, New York, 10016, United States
Carolinas Medical Center
Charlotte, North Carolina, 28203, United States
Duke Cardiology at Southpoint
Durham, North Carolina, 27713, United States
University of Cincinnati Medical Center
Cincinnati, Ohio, 45219, United States
Oregon Health and Science University
Portland, Oregon, 97239, United States
St. Luke's Cardiology Associates
Bethlehem, Pennsylvania, 18018, United States
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
UPMC Presbyterian
Pittsburgh, Pennsylvania, 15213, United States
The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390-9034, United States
Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic
Houston, Texas, 77030, United States
Houston Methodist Hospital
Houston, Texas, 77030, United States
Intermountain Medical Center
Murray, Utah, 84107, United States
University of Utah Medical Center
Salt Lake City, Utah, 84132, United States
University of Virginia
Charlottesville, Virginia, 22908, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
University of Washington Medical Center
Seattle, Washington, 98195, United States
Unity Point Health Meriter Heart and Vascular Institute
Madison, Wisconsin, 53713, United States
Related Publications (1)
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.
PMID: 32466879DERIVED
MeSH Terms
Interventions
Results Point of Contact
- Title
- Bristol-Myers Squibb Study Director
- Organization
- Bristol-Myers Squibb
Study Officials
- STUDY DIRECTOR
Myokardia Medical Information Team
MyoKardia, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 9, 2018
First Posted
February 22, 2018
Study Start
March 30, 2018
Primary Completion
January 7, 2020
Study Completion
January 7, 2020
Last Updated
August 9, 2022
Results First Posted
August 9, 2022
Record last verified: 2022-07