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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)

Deliver Insights in Hypertrophic Cardiomyopathy and Observational Outcomes in Real World: United States and European Prospective Registry Study

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
18 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

Not stated in the study description — ask the study team about visits and procedures.

Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study follows adults with obstructive HCM, a condition where thick heart muscle blocks blood flow and causes symptoms. It tracks usual treatments and health over time in the United States and Europe, including people whose past treatments did not work or caused problems. It looks at mavacamten's safety in both regions and how well it works in Europe.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Be age 18 or older.
  • Give written permission to join and any required permission to use health information. A legal representative may give permission if needed.
  • Have obstructive HCM diagnosed using the heart care guidelines for your region.
  • US: Have heart muscle thickness of at least 15 mm, or 13 mm with a family history of HCM, not explained only by another condition. The heart's main pumping chamber must not be enlarged, and pressure across its blood exit must be at least 30 mmHg.
  • Have a heart ultrasound showing that the heart pumps out at least 55% of its blood with each beat. In the US, this test must be from the past 6 months.
  • US: Have symptoms rated NYHA class II–IV, meaning symptoms that limit activity or occur at rest.
  • Europe: Have symptoms rated NYHA class II–III, meaning some or major limits on activity. These must be recorded at joining or, if unavailable then, within the prior 6 months.
  • Receive usual HCM medicines: beta blockers, certain calcium channel blockers, or disopyramide; receive mavacamten in the US or start it when joining in Europe; or receive no treatment because past treatment failed or caused problems.

Who can't join

  • Have a disease that looks like HCM, such as Fabry disease or amyloidosis, or thick heart muscle linked to high blood pressure.
  • Have a fixed blockage where blood leaves the heart, such as a narrowed or replaced aortic valve.
  • Had surgery or an alcohol procedure to reduce thick heart muscle within the past 6 months. An unsuccessful procedure more than 6 months ago may be allowed.
  • Have never taken beta blockers, certain calcium channel blockers, or disopyramide for obstructive HCM.
  • US: Receive an experimental HCM drug in a treatment trial when joining, or have ever joined a long-term mavacamten safety extension study.
  • Europe: Receive an experimental HCM drug or a drug that targets the heart muscle protein myosin when joining.
  • Europe: Have ever joined another HCM registry or a mavacamten study.
  • Europe: Have taken mavacamten before.

Simplified for readability. The official criteria below are what the study team uses.

Official 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

Study sites (60)

AK

AR

AZ

CA

CO

CT

DC

FL

HI

IA

IL

IN

MA

MD

MI

MN

MO

MS

NC

NE

  • CHI Health Reseach Center · Omaha, NE 68124Amjad Kabach, Site 0070 · Study contactMore trials in Omaha

NJ

NY

OH

PA

SC

TN

TX

VA

WA

WI

WV