A Prospective Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive-HCM)
- Conditions
- Obstructive Hypertrophic Cardiomyopathy
- Interventions
- Drug: Non-mavacamten symptomatic oHCM therapy
- Registration Number
- NCT05489705
- Lead Sponsor
- Bristol-Myers Squibb
- Brief Summary
This registry evaluates patient characteristics, real-world treatment patterns, and short- and long-term outcomes in a population of patients in the United States and Europe with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who are receiving mavacamten, receiving other treatment for obstructive HCM, or not receiving treatment for obstructive HCM due to intolerance or failure of prior treatment.
United States Sub-Study: The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.
Europe Sub-Study: The purpose of this study is to evaluate the effectiveness and safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 1600
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Beta-blocker (BB) / non-dihydropyridine (non-DHP) calcium channel blocker (CCB) / disopyramide Non-mavacamten symptomatic oHCM therapy Participants will receive BB/non-DHP CCB/disopyramide as prescribed by a physician according to standard of care for symptomatic oHCM Mavacamten Mavacamten Participants will receive mavacamten as prescribed by a physician according to standard of care for symptomatic oHCM
- Primary Outcome Measures
Name Time Method Change in resting left ventricular outflow tract (LVOT) gradient from baseline Up to 18 months European Participants only
Incidence rate of new or worsening heart failure due to systolic dysfunction among participants with symptomatic obstructive HCM during periods of exposure to mavacamten and other oHCM treatment Up to 5 Years Systolic dysfunction defined as symptomatic Left Ventricular Ejection Fraction (LVEF) \< 50%. United States participants only.
- Secondary Outcome Measures
Name Time Method New or worsening heart failure due to systolic dysfunction Baseline and up to 18 months Symptomatic left ventricular ejection fraction (LVEF) \<50%. European Participants only
Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 50 mmHG Baseline and up to 18 months European participants only
Evaluation of patient reported outcome measure: Kansas City Cardiomyopathy Questionnaire 23 (KCCQ-23) Baseline, and up to 5 Years The KCCQ-23 is a disease-specific health status instrument composed of 23 items that quantifies the domains of physical limitations, symptoms, self-efficacy, social limitation and quality of life from heart failure. Scores range from 0-100, in which higher scores reflect better health status. United States participants only
Evaluation of patient reported outcome measure: EuroQol Five Dimensions Questionnaire (5-level) (EQ-5D-5L): Health Utility Index Baseline, and up to 5 Years The EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L health utility index and EQ Visual Analog Scale (VAS). EQ-5D-5L health utility index is comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each has 5 levels of perceived problems (1-no problem, 2-slight problems, 3-moderate problems, 4-severe problems, 5-extreme problems). Participants select an answer for each of the 5 dimensions that best matches his/her health "today". Responses are used to generate a weighted summary index, which ranges from 0 to 1.00. A higher score indicates better health and positive changes from baseline indicate improvement of health. United States participants only
Evaluation of patient reported outcome measure: EQ-5D-5L: VAS Baseline, and up to 5 Years The EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and VAS.
The VAS self-rating records the participant's own assessment of his or her overall health status at the time of completion, on a vertical line VAS with scale of 0 (the worst health you can imagine) to 100 (the best health you can imagine). A higher score indicates better health and positive changes from baseline indicate improvement of health status. United States participants onlyOccurrence of arrhythmia Baseline, and up to 18 months (European participants), or 5 Years (United States participants) Incidence rates of participants with arrhythmia (new onset)
Occurrence of non-fatal acute Myocardial Infarction (MI) Up to 5 Years United States participants only
Occurrence of all-cause mortality Baseline, and up to 18 months (European participants), or 5 Years (United States participants) Evaluation of biomarkers of response: Cardiac troponin Baseline, and up to 5 Years Change From Baseline in Serum Concentration of Cardiac Troponins. United States participants only
Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 30 mmHG Baseline and up to 18 months European participants only
Occurrence of Major Adverse Cardiovascular Events (MACE) Up to 5 Years A composite endpoint consisting of non-fatal acute Myocardial Infarction (MI), stroke, hospitalization due to Heart Failure (HF) and Cardiovascular (CV) mortality. United States participants only.
Occurrence of Stroke Up to 5 Years United States participants only
Occurrence of hospitalization due to heart failure Baseline, and up to 18 months (European participants), or 5 Years (United States participants) Occurrence of cardiovascular mortality Up to 5 Years United States participants only
Evaluation of functional responses: New York Heart Association (NYHA) function class Baseline, and up to 18 months (European participants), or 5 Years (United States participants) Percentage of Participants Whose NYHA Class Changes
Evaluation of functional responses: Left Ventricular Outflow Tract (LVOT) gradient Baseline, and up to 18 months (European participants), or 5 Years (United States participants) Mean Change From Baseline in LVOT Gradients At Rest and Provoked
Evaluation of functional responses: LVEF Baseline, and up to 18 months (European participants), or 5 Years (United States participants) Mean Change from Baseline in LVEF
Evaluation of biomarkers of response: NT-proBNP Baseline, and up to 5 Years Change From Baseline in Serum concentration of NT-proBNP. United States participants only
Trial Locations
- Locations (98)
Alaska Heart Institute
🇺🇸Anchorage, Alaska, United States
Pima Heart and Vascular
🇺🇸Tucson, Arizona, United States
UAMS
🇺🇸Little Rock, Arkansas, United States
UC San Diego School of Medicine
🇺🇸La Jolla, California, United States
Keck School of Medicine of USC-Usc
🇺🇸Los Angeles, California, United States
Local Institution - 0088
🇦🇹Innsbruck, Austria
Stanford Health Care Hospital & Clinics
🇺🇸Palo Alto, California, United States
University Of California San Francisco Medical Center
🇺🇸San Francisco, California, United States
UC Denver, AMC
🇺🇸Aurora, Colorado, United States
Hartford HealthCare
🇺🇸Hartford, Connecticut, United States
MedStar Washington Hospital Center
🇺🇸Washington, District of Columbia, United States
Uf Health Jacksonville
🇺🇸Jacksonville, Florida, United States
University of Hawaii Cancer Center
🇺🇸Honolulu, Hawaii, United States
University of Chicago Dept of Medicine
🇺🇸Chicago, Illinois, United States
Indiana. University
🇺🇸Indianapolis, Indiana, United States
Franciscan Physician Network-Indiana Heart Physicians
🇺🇸Indianapolis, Indiana, United States
University of Iowa Hospitals & Clinics
🇺🇸Iowa City, Iowa, United States
University Of Kansas Medical Center & Medical Pavilion
🇺🇸Kansas City, Kansas, United States
University Of Maryland, Ihv
🇺🇸Baltimore, Maryland, United States
MedStar Health Research Institute
🇺🇸Baltimore, Maryland, United States
Harvard (Massachusetts General Hospital)
🇺🇸Boston, Massachusetts, United States
Harvard Medical School - Brigham and Women's Hospital (BWH)
🇺🇸Boston, Massachusetts, United States
Spectrum Health Medical Group
🇺🇸Grand Rapids, Michigan, United States
Henry Ford Health System
🇺🇸West Bloomfield, Michigan, United States
Minneapolis Heart Institute Foundation
🇺🇸Minneapolis, Minnesota, United States
Cardiology Associates Research, Llc
🇺🇸Tupelo, Mississippi, United States
St. Luke's Mid-America Heart Institute
🇺🇸Kansas City, Missouri, United States
Washington University
🇺🇸Saint Louis, Missouri, United States
CHI Health Reseach Center
🇺🇸Omaha, Nebraska, United States
Hackensack University Medical Center
🇺🇸Hackensack, New Jersey, United States
Morristown Medical Center
🇺🇸Morristown, New Jersey, United States
Bassett Medical Center
🇺🇸Cooperstown, New York, United States
Northwell Health
🇺🇸Manhasset, New York, United States
Mount Sinai West
🇺🇸New York, New York, United States
Mount Sinai Hospital
🇺🇸New York, New York, United States
Saint Francis Hospital
🇺🇸Roslyn, New York, United States
WMCHealth Advanced Physician Services
🇺🇸Valhalla, New York, United States
Focus Clinical Research
🇺🇸Charlotte, North Carolina, United States
Duke University Health System
🇺🇸Durham, North Carolina, United States
Christ Hospital Health Network
🇺🇸Cincinnati, Ohio, United States
University Hospitals Cleveland Medical Center
🇺🇸Cleveland, Ohio, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
St. Luke's University Health Network
🇺🇸Bethlehem, Pennsylvania, United States
Lancaster General Hospital
🇺🇸Lancaster, Pennsylvania, United States
AHN Allegheny General Hospital
🇺🇸Pittsburgh, Pennsylvania, United States
University Of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Geisinger Health System
🇺🇸Wilkes-Barre, Pennsylvania, United States
Prisma Health - Upstate
🇺🇸Greenville, South Carolina, United States
Tristar Centennial Medical Ctr
🇺🇸Nashville, Tennessee, United States
Saint Thomas Health
🇺🇸Nashville, Tennessee, United States
North Texas Cardiology Center
🇺🇸Dallas, Texas, United States
Baylor Scott & White Research Institute
🇺🇸Dallas, Texas, United States
The Methodist Hospital, Methodist Cancer Cencer
🇺🇸Houston, Texas, United States
BI Research Center
🇺🇸Houston, Texas, United States
Baylor Scott & White The Heart Hospital Plano
🇺🇸Plano, Texas, United States
Baylor Scott & White Medical Centre - Temple
🇺🇸Temple, Texas, United States
The Rector and Visitors of the Univ of Virginia
🇺🇸Charlottesville, Virginia, United States
WVU Hospitals
🇺🇸Morgantown, West Virginia, United States
University Of Wisconsin - Madison
🇺🇸Madison, Wisconsin, United States
Medical College of Wisconsin, Inc (PI Address)
🇺🇸Milwaukee, Wisconsin, United States
Local Institution - 0082
🇦🇹Braunau am Inn, Austria
Local Institution - 0084
🇦🇹Klagenfurt am Worthersee, Austria
VCU Medical Center
🇺🇸Richmond, Virginia, United States
Carilion Clinic; Virginia Tech-Carilion School of Medicine
🇺🇸Roanoke, Virginia, United States
Virginia Mason Medical Center
🇺🇸Seattle, Washington, United States
Local Institution - 0087
🇦🇹Graz, Austria
Local Institution - 0086
🇦🇹Linz, Austria
Local Institution - 0085
🇦🇹St. Pölten, Austria
Local Institution - 0083
🇦🇹Wien, Austria
Centre Hospitalier Haguenau
🇫🇷Haguenau, Bas Rhin, France
Local Institution - 0093
🇫🇷Saint-Herblain, Loire Atlantique, France
Local Institution - 0090
🇫🇷Lille, Nord, France
Local Institution - 0091
🇫🇷Amiens Cedex 1, Somme, France
Local Institution - 0094
🇫🇷Toulon, Var, France
Local Institution - 0089
🇫🇷Poitiers, Vienne, France
Local Institution - 0098
🇩🇪Aalen, Baden Wuerttemberg, Germany
Local Institution - 0101
🇩🇪Erlangen, Bayern, Germany
Local Institution - 0095
🇩🇪Bernau, Berlin, Germany
Local Institution - 0100
🇩🇪Muenster, Nordrhein Westfalen, Germany
Local Institution - 0099
🇩🇪Dresden, Sachsen, Germany
Local Institution - 0097
🇩🇪Leipzig, Sachsen, Germany
Local Institution - 0096
🇩🇪Berlin, Germany
Research & Cardiovascular Corp
🇵🇷Ponce, Puerto Rico
Local Institution - 0106
🇪🇸Oviedo, Asturias, Spain
Local Institution - 0109
🇪🇸Palma De Mallorca, Baleares, Spain
Local Institution - 0104
🇪🇸Majadahonda, Madrid, Spain
Local Institution - 0105
🇪🇸El Palmar, Murcia, Spain
Local Institution - 0107
🇪🇸Burgos, Spain
Local Institution - 0102
🇪🇸Madrid, Spain
Local Institution - 0108
🇪🇸Madrid, Spain
Local Institution - 0103
🇪🇸Malaga, Spain
Local Institution - 0113
🇬🇧London, Greater London, United Kingdom
Local Institution - 0115
🇬🇧London, Greater London, United Kingdom
Local Institution - 0112
🇬🇧Manchester, Greater Manchester, United Kingdom
Local Institution - 0111
🇬🇧Liverpool, Merseyside, United Kingdom
Local Institution - 0110
🇬🇧Birmingham, West Midlands, United Kingdom
Local Institution - 0114
🇬🇧Leeds, West Yorkshire, United Kingdom
Local Institution - 0116
🇬🇧Cardiff, United Kingdom