Recruiting
Observational
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)
Condition(s) studied
Obstructive Hypertrophic Cardiomyopathy
Investigational drug(s) / intervention(s)
MavacamtenNon-mavacamten symptomatic oHCM therapy
Mavacamten: As per product label
Non-mavacamten symptomatic oHCM therapy: Including Beta-blocker (BB), non-dihydropyridine (non-DHP) calcium channel blocker (CCB) and/or disopyramide as per product label
Study 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.
Eligibility
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
Primary outcome measure(s)
- 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.
- Change in resting left ventricular outflow tract (LVOT) gradient from baseline — Up to 18 months
European Participants only
Trial sites (101)
| Facility | City | Region | Status |
| Alaska Heart Institute |
Anchorage |
Alaska |
Recruiting |
| Pima Heart and Vascular |
Tucson |
Arizona |
Recruiting |
| UAMS |
Little Rock |
Arkansas |
Recruiting |
| Local Institution - 0037 |
La Jolla |
California |
Active Not Recruiting |
| Keck School of Medicine of USC-Usc |
Los Angeles |
California |
Recruiting |
| Stanford Health Care Hospital & Clinics |
Palo Alto |
California |
Recruiting |
| University Of California San Francisco Medical Center |
San Francisco |
California |
Recruiting |
| UC Denver, AMC |
Aurora |
Colorado |
Recruiting |
| Local Institution - 0067 |
Hartford |
Connecticut |
Active Not Recruiting |
| MedStar Washington Hospital Center |
Washington D.C. |
District of Columbia |
Recruiting |
| Local Institution - 0065 |
Jacksonville |
Florida |
Active Not Recruiting |
| Local Institution - 0046 |
Honolulu |
Hawaii |
Active Not Recruiting |
| University of Chicago Dept of Medicine |
Chicago |
Illinois |
Recruiting |
| Indiana. University |
Indianapolis |
Indiana |
Recruiting |
| Franciscan Physician Network-Indiana Heart Physicians |
Indianapolis |
Indiana |
Recruiting |
| Local Institution - 0045 |
Iowa City |
Iowa |
Active Not Recruiting |
| Local Institution - 0059 |
Baltimore |
Maryland |
Active Not Recruiting |
| MedStar Health Research Institute |
Baltimore |
Maryland |
Recruiting |
| Harvard (Massachusetts General Hospital) |
Boston |
Massachusetts |
Recruiting |
| Harvard Medical School - Brigham and Women's Hospital (BWH) |
Boston |
Massachusetts |
Recruiting |
| Spectrum Health Medical Group |
Grand Rapids |
Michigan |
Recruiting |
| Henry Ford Health System |
West Bloomfield |
Michigan |
Recruiting |
| Minneapolis Heart Institute Foundation |
Minneapolis |
Minnesota |
Recruiting |
| Cardiology Associates Research, Llc |
Tupelo |
Mississippi |
Recruiting |
| St. Luke's Mid-America Heart Institute |
Kansas City |
Missouri |
Recruiting |
| Local Institution - 0033 |
Kansas City |
Missouri |
Active Not Recruiting |
| Washington University |
St Louis |
Missouri |
Recruiting |
| CHI Health Reseach Center |
Omaha |
Nebraska |
Recruiting |
| Hackensack University Medical Center |
Hackensack |
New Jersey |
Recruiting |
| Local Institution - 0026 |
Morristown |
New Jersey |
Active Not Recruiting |
| Bassett Medical Center |
Cooperstown |
New York |
Recruiting |
| Local Institution - 0009 |
New York |
New York |
Active Not Recruiting |
| Mount Sinai Hospital |
New York |
New York |
Recruiting |
| Saint Francis Hospital |
Roslyn |
New York |
Recruiting |
| WMCHealth Advanced Physician Services |
Valhalla |
New York |
Recruiting |
| Focus Clinical Research |
Charlotte |
North Carolina |
Recruiting |
| Duke University Health System |
Durham |
North Carolina |
Recruiting |
| Christ Hospital Health Network |
Cincinnati |
Ohio |
Recruiting |
| University Hospitals Cleveland Medical Center |
Cleveland |
Ohio |
Recruiting |
| Local Institution - 0003 |
Cleveland |
Ohio |
Active Not Recruiting |
+ 61 more sites — see the full list on the official registry below.