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
Status: Completed
Location: See all (32) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• 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
Locations
United States
Arizona
Mayo Clinic Arizona
Scottsdale
California
Cedars-Sinai Medical Center (Smidt Heart Institute)
Los Angeles
Stanford Hospital and Clinics/Stanford University
Palo Alto
University of California, San Francisco
San Francisco
Connecticut
Yale New Haven Hospital
New Haven
Iowa
University of Iowa Hospitals and clinics
Iowa City
Illinois
Northwestern University
Evanston
Indiana
St. Vincent Medical Group
Indianapolis
Massachusetts
Brigham and Women's Hospital
Boston
Maryland
University of Maryland Medical System
Baltimore
Michigan
Michigan Medicine
Ann Arbor
Henry Ford Hospital
Detroit
Missouri
Washington University School of Medicine
Saint Louis
North Carolina
Carolinas Medical Center
Charlotte
Duke Cardiology at Southpoint
Durham
New York
NYU Winthrop Hospital
Mineola
NYU Langone Medical Center
New York
Ohio
University of Cincinnati Medical Center
Cincinnati
Oregon
Oregon Health and Science University
Portland
Pennsylvania
St. Luke's Cardiology Associates
Bethlehem
Penn State Health Milton S. Hershey Medical Center
Hershey
University of Pennsylvania
Philadelphia
UPMC Presbyterian
Pittsburgh
Texas
The University of Texas Southwestern Medical Center at Dallas
Dallas
Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic
Houston
Houston Methodist Hospital
Houston
Utah
Intermountain Medical Center
Murray
University of Utah Medical Center
Salt Lake City
Virginia
University of Virginia
Charlottesville
Virginia Commonwealth University
Richmond
Washington
University of Washington Medical Center
Seattle
Wisconsin
Unity Point Health Meriter Heart and Vascular Institute
Madison
Time Frame
Start Date: 2018-03-30
Completion Date: 2020-01-07
Participants
Target number of participants: 59
Treatments
Experimental: Group 1
Active Treatment for participants with base target trough concentration
Experimental: Group 2
Active Treatment for participants with higher target trough concentration
Placebo_comparator: Placebo
Placebo Group
Authors
Aslan Turer, Paulino Alvarez, John Symanski, Ali Marian, Timothy Wong, Neal Lakdawala, Ashwin Ravichandran, Daniel Jacoby, Matthew Wheeler, Christopher Kramer, Mark Sherrid, Florian Rader, Keyur Shah, Kia Afshar, Theodore Abraham, Anjali Owens, Karthikeyan Ananthasubramaniam, Ramesh Daggubati, Stephen Heitner, Sharlene Day, Sherif Nagueh, Jamshid Shirani, Eric Popjes, Richard Bach, David Owens, Lubna Choudhury, Andrew Wang, Libin Wang, John Moses
Related Therapeutic Areas
Sponsors
Leads: MyoKardia, Inc.