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)

Launched by BRISTOL-MYERS SQUIBB · Aug 3, 2022

Keywords

ClinConnect Summary

This clinical trial is studying the real-world characteristics and treatment outcomes of patients with symptomatic obstructive hypertrophic cardiomyopathy (Obstructive-HCM), a condition where the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. The trial focuses on patients in the United States and Europe who are receiving mavacamten, a new medication for this condition, as well as those receiving other treatments or no treatment due to previous failures or side effects. By collecting data on how these patients respond to their treatments over time, researchers hope to better understand the safety and effectiveness of mavacamten in everyday clinical settings.

To be eligible for this study, participants must be at least 18 years old and have a documented diagnosis of obstructive HCM that meets specific medical guidelines. They should also have certain heart function measurements and be experiencing symptoms that affect their daily activities. Participants can expect to share information about their health and treatment experiences, which will help researchers gather valuable insights. This study is currently recruiting participants, and it's important to note that those who are already part of other similar studies or have been treated with certain other medications may not be eligible.

Gender

ALL

Eligibility criteria

• • 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