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)

Recruiting

• Conditions: Obstructive Hypertrophic Cardiomyopathy
• Interventions: Drug: Non-mavacamten symptomatic oHCM therapy; Drug: Mavacamten

• 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

Study Timeline

• Study First Submitted: 2022-08-03
• Study First Submitted QC: 2022-08-03
• Study First Posted: 2022-08-05
• Study Start: 2022-08-16
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-10
• Last Update Posted: 2025-04-11
• Primary Completion: 2029-08-17
• Study Completion: 2029-08-17

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 1600

Inclusion Criteria

Not provided

Exclusion Criteria

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 only
Occurrence 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

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

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