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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)
Not Recruiting
Trial ID: NCT05489705
Purpose
The purpose of this study is to evaluate the safety of mavacamten in patients with
symptomatic obstructive hypertrophic cardiomyopathy (HCM) treated in the real-world setting.
The registry study also provide a real-world understanding of the current obstructive HCM
patient population, treatment patterns, and clinical relevant outcomes for patients with
symptomatic obstructive HCM in the US.
Official Title
Deliver Insights in Hypertrophic Cardiomyopathy and Observational Outcomes in Real World: United States Prospective Registry Study
Stanford Investigator(s)
Matthew Wheeler
Associate Professor of Medicine (Cardiovascular Medicine)
Eligibility
Inclusion Criteria:
1. Willing and able to provide written informed consent form (ICF) and any required
privacy authorization prior to the initiation of study procedures. i. Diagnosis of
obstructive HCM consistent with 2020 American Heart Association/American College of
Cardiology (AHA/ACC) guidelines. ii. 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.
2. Has documented LVEF of ≥ 55% recorded by echocardiography within the last 6 months.
3. Symptoms consistent with NYHA functional class II-IV.
4. ≥ 18 years of age at the time of informed consent.
5. Receiving BBs, non-dihydropyridine calcium. channel blockers (non-DHP 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.
Exclusion Criteria:
1. Known phenocopy disease (eg, Fabry disease, amyloidosis) or LV hypertrophy associated
with hypertension.
2. Documentation of any fixed obstruction of the outflow tract such as aortic valve
stenosis or replacement.
3. 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.
4. Naïve to treatment for obstructive HCM (ie, never treated with BBs, non-DHP CCBs, or
disopyramide).
5. Receiving an investigational therapeutic agent for obstructive HCM (eg,
myosin-inhibitors other than mavacamten) in an interventional clinical trial at
participant enrollment.
6. 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])
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305