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)

Bristol-Myers Squibb1,600 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

1,600

Conditions

Obstructive Hypertrophic Cardiomyopathy

Interventions

MavacamtenDRUG

As per product label

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria * ≥ 18 years of age at the time of informed consent. * Willing and able to provide written informed consent form (ICF) and any required privacy authorization prior to the initiation of study procedures (or in those situations where consent cannot be given by participants, consent provided by their legally acceptable representatives) United States Sub-Study * Diagnosis of obstructive HCM consistent with 2020 American Heart Association/American College of Cardiology (AHA/ACC) guidelines. * Obstructive HCM is defined clinically by the presence of increased LV wall thickness ≥ 15 mm (or ≥ 13 mm with positive family history of HCM) in a nondilated ventricular chamber that is not solely explained by abnormal loading conditions (eg, another cardiac or systemic disease) and peak LVOT gradient of ≥ 30 mmHg at rest or with provocation. * Has documented LVEF of ≥ 55% recorded by echocardiography within the last 6 months. * Symptoms consistent with NYHA functional class II-IV. * Receiving beta blocker (BB)s, non-dihydropyridine calcium. channel blockers (nonDHP CCBs), disopyramide, and/or mavacamten (once available) as part of routine clinical care; or currently receiving no treatment due to intolerance or failure of prior treatment (eg, BBs, non-DHP CCBs, or disopyramide) for obstructive HCM. European Sub-study * Diagnosis of obstructive HCM consistent with the most recent European Society of Cardiology (ESC) and American Heart Association/American College of Cardiology (AHA/ACC) guidelines * Documented LVEF of ≥55% recorded by TTE * Documented symptoms consistent with NYHA functional class II-III at enrollment or within 6 months prior to enrollment (if not available at enrollment). * As part of routine clinical care for obstructive HCM: receiving BBs, non-DHP CCBs, disopyramide; initiating mavacamten at enrollment; or currently receiving no treatment due to intolerance or failure of prior treatment (e.g., BBs, non-DHP CCBs, or disopyramide). Exclusion Criteria * Known phenocopy disease (e.g., Fabry disease, amyloidosis) or LV hypertrophy associated with hypertension. * Documentation of any fixed obstruction of the outflow tract such as aortic valve stenosis or replacement. * Prior treatment of obstructive HCM with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation \[ASA\]) within 6 months prior to enrollment; participants with an unsuccessful myectomy or percutaneous ASA performed \> 6 months prior to enrollment may be enrolled. * Naïve to treatment for obstructive HCM (ie, never treated with BBs, nonDHP CCBs, or disopyramide). United States Sub-Study * Receiving an investigational therapeutic agent for obstructive HCM (eg, myosin-inhibitors other than mavacamten) in an interventional clinical trial at participant enrollment. * Previously or currently enrolled in a long-term safety extension study of mavacamten (eg, EXPLORER-HCM \[ClinicalTrials.gov, NCT03470545\], MAVA-LTE \[NCT03723655\], PIONEER-OLE \[NCT03496168\], VALORHCM \[NCT04349072\], or MAVERICK \[NCT03442764\]) European Sub-study * Receiving an investigational therapeutic agent or any cardiac myosin inhibitor and/or modulators for obstructive HCM at patient enrolment * Previously or currently enrolled in other HCM registry studies (e.g., TORCH, REMY, EU-PASS) * Previously or currently enrolled in a study of mavacamten (e.g., EXPLORER-HCM \[ClinicalTrials.gov, NCT03470545\], MAVA-LTE \[NCT03723655\], PIONEER-OLE \[NCT03496168\], VALOR-HCM \[NCT04349072\], MAVERICK \[NCT03442764\], or MEMENTO \[NCT2264899\]) * Previously treated with mavacamten

Locations (99)

Alaska Heart Institute

Anchorage, Alaska, United States

RECRUITING

Pima Heart and Vascular

Tucson, Arizona, United States

RECRUITING

UAMS

Little Rock, Arkansas, United States

RECRUITING

UC San Diego School of Medicine

La Jolla, California, United States

Outcomes

Primary Outcomes

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

Systolic dysfunction defined as symptomatic Left Ventricular Ejection Fraction (LVEF) \< 50%. United States participants only.

Time frame: Up to 5 Years

Change in resting left ventricular outflow tract (LVOT) gradient from baseline

European Participants only

Time frame: Up to 18 months

Secondary Outcomes

Occurrence of arrhythmia

Incidence rates of participants with arrhythmia (new onset)

Time frame: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)

New or worsening heart failure due to systolic dysfunction

Symptomatic left ventricular ejection fraction (LVEF) \<50%. European Participants only

Time frame: Baseline and up to 18 months

Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 50 mmHG

European participants only

Time frame: Baseline and up to 18 months

Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 30 mmHG

European participants only

Time frame: Baseline and up to 18 months

Occurrence of Major Adverse Cardiovascular Events (MACE)

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.

Time frame: Up to 5 Years

Occurrence of non-fatal acute Myocardial Infarction (MI)

United States participants only

Time frame: Up to 5 Years

Occurrence of Stroke

United States participants only

Time frame: Up to 5 Years

Occurrence of hospitalization due to heart failure

Time frame: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)

Occurrence of cardiovascular mortality

United States participants only

Time frame: Up to 5 Years

Occurrence of all-cause mortality

Time frame: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)

Evaluation of functional responses: New York Heart Association (NYHA) function class

Percentage of Participants Whose NYHA Class Changes

Time frame: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)

Evaluation of functional responses: Left Ventricular Outflow Tract (LVOT) gradient

Mean Change From Baseline in LVOT Gradients At Rest and Provoked

Time frame: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)

Evaluation of functional responses: LVEF

Mean Change from Baseline in LVEF

Time frame: Baseline, and up to 18 months (European participants), or 5 Years (United States participants)

Evaluation of patient reported outcome measure: Kansas City Cardiomyopathy Questionnaire 23 (KCCQ-23)

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

Time frame: Baseline, and up to 5 Years

Evaluation of patient reported outcome measure: EuroQol Five Dimensions Questionnaire (5-level) (EQ-5D-5L): Health Utility Index

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

Time frame: Baseline, and up to 5 Years

Evaluation of patient reported outcome measure: EQ-5D-5L: VAS

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

Time frame: Baseline, and up to 5 Years

Evaluation of biomarkers of response: NT-proBNP

Change From Baseline in Serum concentration of NT-proBNP. United States participants only

Time frame: Baseline, and up to 5 Years

Evaluation of biomarkers of response: Cardiac troponin

Change From Baseline in Serum Concentration of Cardiac Troponins. United States participants only

Time frame: Baseline, and up to 5 Years

Central Contacts

BMS Study Connect Contact Center www.BMSStudyConnect.com

CONTACT

855-907-3286 Clinical.Trials@bms.com

First line of the email MUST contain the NCT# and Site #.

CONTACT