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)

NCT05489705 | Recruiting FDA Drug

• 1 other identifier: CV027-012
• Study Type: observational
• Target: 1,600
• Locations: 7 countries
• Sites: 99

Brief 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.

Conditions

Obstructive Hypertrophic Cardiomyopathy

Keywords

Obstructive hypertrophic cardiomyopathy; Obstructive HCM (oHCM); Mavacamten; Heart failure; oHCM

Outcome Measures

Primary Outcomes (2)

• 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.

  Up to 5 Years

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

  European Participants only

  Up to 18 months

Secondary Outcomes (18)

• Occurrence of arrhythmia

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

• New or worsening heart failure due to systolic dysfunction

  Baseline and up to 18 months

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

  Baseline and up to 18 months

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

  Baseline and up to 18 months

• Occurrence of Major Adverse Cardiovascular Events (MACE)

  Up to 5 Years

Study Arms (2)

Mavacamten

Participants will receive mavacamten as prescribed by a physician according to standard of care for symptomatic oHCM

Drug: Mavacamten

Beta-blocker (BB) / non-dihydropyridine (non-DHP) calcium channel blocker (CCB) / disopyramide

Participants will receive BB/non-DHP CCB/disopyramide as prescribed by a physician according to standard of care for symptomatic oHCM

Drug: Non-mavacamten symptomatic oHCM therapy

Interventions

Mavacamten DRUG

As per product label

Mavacamten

Non-mavacamten symptomatic oHCM therapy DRUG

Including Beta-blocker (BB), non-dihydropyridine (non-DHP) calcium channel blocker (CCB) and/or disopyramide as per product label

Beta-blocker (BB) / non-dihydropyridine (non-DHP) calcium channel blocker (CCB) / disopyramide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will include adult participants with symptomatic obstructive HCM who are receiving pharmacological treatment for obstructive HCM (for example, BBs, non-DHP CCBs, disopyramide, and/or mavacamten) as part of routine care in the United States or Europe.

You may qualify if:

• ≥ 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).

You may not qualify if:

• 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

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (99)

Alaska Heart Institute

Anchorage, Alaska, 99508, United States

RECRUITING

Pima Heart and Vascular

Tucson, Arizona, 85704, United States

RECRUITING

UAMS

Little Rock, Arkansas, 72205-7199, United States

RECRUITING

UC San Diego School of Medicine

La Jolla, California, 92037, United States

RECRUITING

Keck School of Medicine of USC-Usc

Los Angeles, California, 90033, United States

RECRUITING

Stanford Health Care Hospital & Clinics

Palo Alto, California, 94304, United States

RECRUITING

University Of California San Francisco Medical Center

San Francisco, California, 94117, United States

RECRUITING

UC Denver, AMC

Aurora, Colorado, 48377, United States

RECRUITING

Hartford HealthCare

Hartford, Connecticut, 06106, United States

RECRUITING

MedStar Washington Hospital Center

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Uf Health Jacksonville

Jacksonville, Florida, 32209, United States

RECRUITING

University of Hawaii Cancer Center

Honolulu, Hawaii, 96813, United States

RECRUITING

University of Chicago Dept of Medicine

Chicago, Illinois, 60637, United States

RECRUITING

Indiana. University

Indianapolis, Indiana, 46202, United States

RECRUITING

Franciscan Physician Network-Indiana Heart Physicians

Indianapolis, Indiana, 46237, United States

RECRUITING

University of Iowa Hospitals & Clinics

Iowa City, Iowa, 52242, United States

RECRUITING

University Of Kansas Medical Center & Medical Pavilion

Kansas City, Kansas, 66160, United States

RECRUITING

University Of Maryland, Ihv

Baltimore, Maryland, 21201, United States

RECRUITING

MedStar Health Research Institute

Baltimore, Maryland, 21218, United States

RECRUITING

Harvard (Massachusetts General Hospital)

Boston, Massachusetts, 02114, United States

RECRUITING

Harvard Medical School - Brigham and Women's Hospital (BWH)

Boston, Massachusetts, 02215, United States

RECRUITING

Spectrum Health Medical Group

Grand Rapids, Michigan, 49525, United States

RECRUITING

Henry Ford Health System

West Bloomfield, Michigan, 48322, United States

RECRUITING

Minneapolis Heart Institute Foundation

Minneapolis, Minnesota, 55407, United States

RECRUITING

Cardiology Associates Research, Llc

Tupelo, Mississippi, 38801, United States

RECRUITING

St. Luke's Mid-America Heart Institute

Kansas City, Missouri, 64111, United States

RECRUITING

Washington University

St Louis, Missouri, 63110, United States

RECRUITING

CHI Health Reseach Center

Omaha, Nebraska, 68124, United States

RECRUITING

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

RECRUITING

Morristown Medical Center

Morristown, New Jersey, 07960, United States

RECRUITING

Bassett Medical Center

Cooperstown, New York, 13326, United States

RECRUITING

Northwell Health

Manhasset, New York, 11030, United States

RECRUITING

Mount Sinai West

New York, New York, 10019, United States

RECRUITING

Mount Sinai Hospital

New York, New York, 10029, United States

RECRUITING

Saint Francis Hospital

Roslyn, New York, 11576, United States

RECRUITING

WMCHealth Advanced Physician Services

Valhalla, New York, 10595, United States

RECRUITING

Focus Clinical Research

Charlotte, North Carolina, 28207, United States

RECRUITING

Duke University Health System

Durham, North Carolina, 27710, United States

RECRUITING

Christ Hospital Health Network

Cincinnati, Ohio, 45219, United States

RECRUITING

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

RECRUITING

Cleveland Clinic

Cleveland, Ohio, 44195, United States

RECRUITING

St. Luke's University Health Network

Bethlehem, Pennsylvania, 18015, United States

RECRUITING

Lancaster General Hospital

Lancaster, Pennsylvania, 17603, United States

RECRUITING

AHN Allegheny General Hospital

Pittsburgh, Pennsylvania, 15212, United States

RECRUITING

University Of Pittsburgh

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

Geisinger Health System

Wilkes-Barre, Pennsylvania, 18711, United States

RECRUITING

Prisma Health - Upstate

Greenville, South Carolina, 29605, United States

RECRUITING

Tristar Centennial Medical Ctr

Nashville, Tennessee, 37203, United States

RECRUITING

Saint Thomas Health

Nashville, Tennessee, 37205-2018, United States

RECRUITING

North Texas Cardiology Center

Dallas, Texas, 75208, United States

RECRUITING

Baylor Scott & White Research Institute

Dallas, Texas, 75246, United States

RECRUITING

The Methodist Hospital, Methodist Cancer Cencer

Houston, Texas, 77030, United States

RECRUITING

BI Research Center

Houston, Texas, 77084, United States

RECRUITING

Baylor Scott & White The Heart Hospital Plano

Plano, Texas, 75093, United States

RECRUITING

Baylor Scott & White Medical Centre - Temple

Temple, Texas, 76508, United States

RECRUITING

The Rector and Visitors of the Univ of Virginia

Charlottesville, Virginia, 22908, United States

RECRUITING

VCU Medical Center

Richmond, Virginia, 23298, United States

RECRUITING

Carilion Clinic; Virginia Tech-Carilion School of Medicine

Roanoke, Virginia, 24014, United States

RECRUITING

Virginia Mason Medical Center

Seattle, Washington, 98101, United States

RECRUITING

WVU Hospitals

Morgantown, West Virginia, 26506, United States

RECRUITING

University Of Wisconsin - Madison

Madison, Wisconsin, 53792, United States

RECRUITING

Medical College of Wisconsin, Inc (PI Address)

Milwaukee, Wisconsin, 53226, United States

RECRUITING

KH St.Josef Braunau am Inn

Braunau am Inn, 5280, Austria

RECRUITING

LKH-Univ. Klinikum Graz

Graz, 8036, Austria

RECRUITING

Local Institution - 0088

Innsbruck, 6020, Austria

NOT YET RECRUITING

Local Institution - 0084

Klagenfurt, 9020, Austria

WITHDRAWN

Kepler Universitätsklinikum Linz

Linz, 4021, Austria

RECRUITING

Local Institution - 0085

Sankt Pölten, 3100, Austria

NOT YET RECRUITING

Local Institution - 0083

Vienna, 1090, Austria

NOT YET RECRUITING

Centre Hospitalier Haguenau

Haguenau, Bas Rhin, 67500, France

RECRUITING

CHU Nantes Hopital Nord Laennec

Saint-Herblain, Loire Atlantique, 44800, France

RECRUITING

Hôpital Saint Vincent-de-Paul

Lille, Nord, 59000, France

RECRUITING

CHU Amiens-Picardie

Amiens, Somme, 80054, France

RECRUITING

Hôpital Sainte Musse

Toulon, Var, 83000, France

RECRUITING

CHU De Poitiers - Hopital De La Miletrie

Poitiers, Vienne, 86000, France

RECRUITING

Local Institution - 0117

Montpellier, 34000, France

WITHDRAWN

Ostalb-Klinikum Aalen

Aalen, Baden-Wurttemberg, 73430, Germany

RECRUITING

Local Institution - 0101

Erlangen, Bavaria, 91054, Germany

WITHDRAWN

Universitatsklinikum Muenster

Münster, North Rhine-Westphalia, 48149, Germany

RECRUITING

Praxisklinik Herz Und Gefasse-Klinikum Weisser Hirsch

Dresden, Saxony, 01099, Germany

RECRUITING

Universitaetsklinikum Leipzig Aoer

Leipzig, Saxony, 04103, Germany

RECRUITING

Immanuel Klinikum Bernau

Bernau, State of Berlin, 16321, Germany

RECRUITING

Kardiologische und angiologische Praxen im Spreebogen

Berlin, 10719, Germany

RECRUITING

Research & Cardiovascular Corp

Ponce, 00717-1322, Puerto Rico

RECRUITING

Hospital Fundacion Son Llatzer

Palma de Mallorca, Balearic Islands, 07198, Spain

RECRUITING

Hospital Universitario Puerta de Hierro Majadahonda

Majadahonda, Madrid, 28222, Spain

RECRUITING

Hospital Universitario Virgen de La Arrixaca

El Palmar, Murcia, 30120, Spain

RECRUITING

Hospital Universitario Central de Asturias

Oviedo, Principality of Asturias, 33011, Spain

RECRUITING

Hospital Universitario de Burgos

Burgos, 09006, Spain

RECRUITING

Local Institution - 0102

Madrid, 28007, Spain

WITHDRAWN

Hospital Clinico San Carlos

Madrid, 28040, Spain

RECRUITING

Hospital Clinico Universitario Virgen de La Victoria

Málaga, 29010, Spain

RECRUITING

Local Institution - 0113

London, Greater London, SW3 6NP, United Kingdom

NOT YET RECRUITING

Imperial College Healthcare NHS Trust

London, Greater London, W6 8RF, United Kingdom

RECRUITING

University Of Manchester

Manchester, Greater Manchester, M13 9WL, United Kingdom

RECRUITING

Liverpool Heart and Chest Hospital

Liverpool, Merseyside, L14 3PE, United Kingdom

RECRUITING

University Hospitals Birmingham NHS Foundation Trust

Birmingham, West Midlands, B15 2GW, United Kingdom

RECRUITING

Leeds General Infirmary

Leeds, West Yorkshire, LS1 3EX, United Kingdom

RECRUITING

University Hospital of Wales

Cardiff, CF14 4XW, United Kingdom

RECRUITING

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic; Heart Failure

Interventions

MYK-461

Condition Hierarchy (Ancestors)

Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Central Study 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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2022
• First Posted: August 5, 2022
• Study Start: August 16, 2022
• Primary Completion (Estimated): August 17, 2029
• Study Completion (Estimated): August 17, 2029
• Last Updated: November 6, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

GB (7); FR (7); US (62); AU (7); DE (7); PR (1); ES (8)