Back to resultsA Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) (MAVERICK-HCM)
Primary Purpose
Non-obstructive Hypertrophic Cardiomyopathy
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
mavacamten
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Non-obstructive Hypertrophic Cardiomyopathy
Eligibility 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.
Sites / Locations
- Mayo Clinic Arizona
- Cedars-Sinai Medical Center (Smidt Heart Institute)
- Stanford Hospital and Clinics/Stanford University
- University of California, San Francisco
- Yale New Haven Hospital
- Northwestern University
- St. Vincent Medical Group
- University of Iowa Hospitals and clinics
- University of Maryland Medical System
- Brigham and Women's Hospital
- Michigan Medicine
- Henry Ford Hospital
- Washington University School of Medicine
- NYU Winthrop Hospital
- NYU Langone Medical Center
- Carolinas Medical Center
- Duke Cardiology at Southpoint
- University of Cincinnati Medical Center
- Oregon Health and Science University
- St. Luke's Cardiology Associates
- Penn State Health Milton S. Hershey Medical Center
- University of Pennsylvania
- UPMC Presbyterian
- The University of Texas Southwestern Medical Center at Dallas
- Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic
- Houston Methodist Hospital
- Intermountain Medical Center
- University of Utah Medical Center
- University of Virginia
- Virginia Commonwealth University
- University of Washington Medical Center
- Unity Point Health Meriter Heart and Vascular Institute
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
Group 1
Group 2
Placebo
Arm Description
Active Treatment for participants with base target trough concentration
Active Treatment for participants with higher target trough concentration
Placebo Group
Outcomes
Primary Outcome Measures
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)
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)
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03442764
Brief Title
A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)
Acronym
MAVERICK-HCM
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
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Completed
Study Start Date
March 30, 2018 (Actual)
Primary Completion Date
January 7, 2020 (Actual)
Study Completion Date
January 7, 2020 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MyoKardia, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Non-obstructive Hypertrophic Cardiomyopathy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
59 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Group 1
Arm Type
Experimental
Arm Description
Active Treatment for participants with base target trough concentration
Arm Title
Group 2
Arm Type
Experimental
Arm Description
Active Treatment for participants with higher target trough concentration
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo Group
Intervention Type
Drug
Intervention Name(s)
mavacamten
Other Intervention Name(s)
MYK-461
Intervention Description
MYK-461
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)
Description
This is the 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)
Title
Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE)
Description
This is the 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)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Myokardia Medical Information Team
Organizational Affiliation
MyoKardia, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Mayo Clinic Arizona
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
Cedars-Sinai Medical Center (Smidt Heart Institute)
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Stanford Hospital and Clinics/Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
University of California, San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Yale New Haven Hospital
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06520
Country
United States
Facility Name
Northwestern University
City
Evanston
State/Province
Illinois
ZIP/Postal Code
60208
Country
United States
Facility Name
St. Vincent Medical Group
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46260
Country
United States
Facility Name
University of Iowa Hospitals and clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Maryland Medical System
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21201
Country
United States
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Michigan Medicine
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Henry Ford Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
NYU Winthrop Hospital
City
Mineola
State/Province
New York
ZIP/Postal Code
11501
Country
United States
Facility Name
NYU Langone Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Carolinas Medical Center
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28203
Country
United States
Facility Name
Duke Cardiology at Southpoint
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27713
Country
United States
Facility Name
University of Cincinnati Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
St. Luke's Cardiology Associates
City
Bethlehem
State/Province
Pennsylvania
ZIP/Postal Code
18018
Country
United States
Facility Name
Penn State Health Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033-0850
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
UPMC Presbyterian
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
The University of Texas Southwestern Medical Center at Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-9034
Country
United States
Facility Name
Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Houston Methodist Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Intermountain Medical Center
City
Murray
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
Facility Name
University of Utah Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Virginia
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
University of Washington Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Unity Point Health Meriter Heart and Vascular Institute
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53713
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
32466879
Citation
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.
Results Reference
derived
Learn more about this trial
A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)
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