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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)

NCT05489705 · Bristol-Myers Squibb
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Official title
Deliver Insights in Hypertrophic Cardiomyopathy and Observational Outcomes in Real World: United States and European Prospective Registry Study
About this study
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 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 design
Enrollment target: 1600 participants
Age groups: adult, older_adult
Timeline
Starts: 2022-08-16
Estimated completion: 2029-08-17
Last updated: 2025-11-06
Interventions
Drug: MavacamtenDrug: Non-mavacamten symptomatic oHCM therapy
Primary outcomes
  • 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)
  • Change in resting left ventricular outflow tract (LVOT) gradient from baseline (Up to 18 months)
Sponsor
Bristol-Myers Squibb · industry
Contacts & investigators
ContactBMS Study Connect Contact Center www.BMSStudyConnect.com · contact · Clinical.Trials@bms.com · 855-907-3286
ContactFirst line of the email MUST contain the NCT# and Site #. · contact
InvestigatorBristol-Myers Squibb · study_director, Bristol-Myers Squibb
All locations (99)
Alaska Heart InstituteRecruiting
Anchorage, Alaska, United States
Pima Heart and VascularRecruiting
Tucson, Arizona, United States
UAMSRecruiting
Little Rock, Arkansas, United States
UC San Diego School of MedicineRecruiting
La Jolla, California, United States
Keck School of Medicine of USC-UscRecruiting
Los Angeles, California, United States
Stanford Health Care Hospital & ClinicsRecruiting
Palo Alto, California, United States
University Of California San Francisco Medical CenterRecruiting
San Francisco, California, United States
UC Denver, AMCRecruiting
Aurora, Colorado, United States
Hartford HealthCareRecruiting
Hartford, Connecticut, United States
MedStar Washington Hospital CenterRecruiting
Washington D.C., District of Columbia, United States
Uf Health JacksonvilleRecruiting
Jacksonville, Florida, United States
University of Hawaii Cancer CenterRecruiting
Honolulu, Hawaii, United States
University of Chicago Dept of MedicineRecruiting
Chicago, Illinois, United States
Indiana. UniversityRecruiting
Indianapolis, Indiana, United States
Franciscan Physician Network-Indiana Heart PhysiciansRecruiting
Indianapolis, Indiana, United States
University of Iowa Hospitals & ClinicsRecruiting
Iowa City, Iowa, United States
University Of Kansas Medical Center & Medical PavilionRecruiting
Kansas City, Kansas, United States
University Of Maryland, IhvRecruiting
Baltimore, Maryland, United States
MedStar Health Research InstituteRecruiting
Baltimore, Maryland, United States
Harvard (Massachusetts General Hospital)Recruiting
Boston, Massachusetts, United States
Harvard Medical School - Brigham and Women's Hospital (BWH)Recruiting
Boston, Massachusetts, United States
Spectrum Health Medical GroupRecruiting
Grand Rapids, Michigan, United States
Henry Ford Health SystemRecruiting
West Bloomfield, Michigan, United States
Minneapolis Heart Institute FoundationRecruiting
Minneapolis, Minnesota, United States
Cardiology Associates Research, LlcRecruiting
Tupelo, Mississippi, United States
St. Luke's Mid-America Heart InstituteRecruiting
Kansas City, Missouri, United States
Washington UniversityRecruiting
St Louis, Missouri, United States
CHI Health Reseach CenterRecruiting
Omaha, Nebraska, United States
Hackensack University Medical CenterRecruiting
Hackensack, New Jersey, United States
Morristown Medical CenterRecruiting
Morristown, New Jersey, United States
Bassett Medical CenterRecruiting
Cooperstown, New York, United States
Northwell HealthRecruiting
Manhasset, New York, United States
Mount Sinai WestRecruiting
New York, New York, United States
Mount Sinai HospitalRecruiting
New York, New York, United States
Saint Francis HospitalRecruiting
Roslyn, New York, United States
WMCHealth Advanced Physician ServicesRecruiting
Valhalla, New York, United States
Focus Clinical ResearchRecruiting
Charlotte, North Carolina, United States
Duke University Health SystemRecruiting
Durham, North Carolina, United States
Christ Hospital Health NetworkRecruiting
Cincinnati, Ohio, United States
University Hospitals Cleveland Medical CenterRecruiting
Cleveland, Ohio, United States
Cleveland ClinicRecruiting
Cleveland, Ohio, United States
St. Luke's University Health NetworkRecruiting
Bethlehem, Pennsylvania, United States
Lancaster General HospitalRecruiting
Lancaster, Pennsylvania, United States
AHN Allegheny General HospitalRecruiting
Pittsburgh, Pennsylvania, United States
University Of PittsburghRecruiting
Pittsburgh, Pennsylvania, United States
Geisinger Health SystemRecruiting
Wilkes-Barre, Pennsylvania, United States
Prisma Health - UpstateRecruiting
Greenville, South Carolina, United States
Tristar Centennial Medical CtrRecruiting
Nashville, Tennessee, United States
Saint Thomas HealthRecruiting
Nashville, Tennessee, United States
North Texas Cardiology CenterRecruiting
Dallas, Texas, United States
Baylor Scott & White Research InstituteRecruiting
Dallas, Texas, United States
The Methodist Hospital, Methodist Cancer CencerRecruiting
Houston, Texas, United States
BI Research CenterRecruiting
Houston, Texas, United States
Baylor Scott & White The Heart Hospital PlanoRecruiting
Plano, Texas, United States
Baylor Scott & White Medical Centre - TempleRecruiting
Temple, Texas, United States
The Rector and Visitors of the Univ of VirginiaRecruiting
Charlottesville, Virginia, United States
VCU Medical CenterRecruiting
Richmond, Virginia, United States
Carilion Clinic; Virginia Tech-Carilion School of MedicineRecruiting
Roanoke, Virginia, United States
Virginia Mason Medical CenterRecruiting
Seattle, Washington, United States
WVU HospitalsRecruiting
Morgantown, West Virginia, United States
University Of Wisconsin - MadisonRecruiting
Madison, Wisconsin, United States
Medical College of Wisconsin, Inc (PI Address)Recruiting
Milwaukee, Wisconsin, United States
KH St.Josef Braunau am InnRecruiting
Braunau am Inn, Austria
LKH-Univ. Klinikum GrazRecruiting
Graz, Austria
Local Institution - 0088Not Yet Recruiting
Innsbruck, Austria
Local Institution - 0084Withdrawn
Klagenfurt, Austria
Kepler Universitätsklinikum LinzRecruiting
Linz, Austria
Local Institution - 0085Not Yet Recruiting
Sankt Pölten, Austria
Local Institution - 0083Not Yet Recruiting
Vienna, Austria
Centre Hospitalier HaguenauRecruiting
Haguenau, Bas Rhin, France
CHU Nantes Hopital Nord LaennecRecruiting
Saint-Herblain, Loire Atlantique, France
Hôpital Saint Vincent-de-PaulRecruiting
Lille, Nord, France
CHU Amiens-PicardieRecruiting
Amiens, Somme, France
Hôpital Sainte MusseRecruiting
Toulon, Var, France
CHU De Poitiers - Hopital De La MiletrieRecruiting
Poitiers, Vienne, France
Local Institution - 0117Withdrawn
Montpellier, France
Ostalb-Klinikum AalenRecruiting
Aalen, Baden-Wurttemberg, Germany
Local Institution - 0101Withdrawn
Erlangen, Bavaria, Germany
Universitatsklinikum MuensterRecruiting
Münster, North Rhine-Westphalia, Germany
Praxisklinik Herz Und Gefasse-Klinikum Weisser HirschRecruiting
Dresden, Saxony, Germany
Universitaetsklinikum Leipzig AoerRecruiting
Leipzig, Saxony, Germany
Immanuel Klinikum BernauRecruiting
Bernau, State of Berlin, Germany
Kardiologische und angiologische Praxen im SpreebogenRecruiting
Berlin, Germany
Research & Cardiovascular CorpRecruiting
Ponce, Puerto Rico
Hospital Fundacion Son LlatzerRecruiting
Palma de Mallorca, Balearic Islands, Spain
Hospital Universitario Puerta de Hierro MajadahondaRecruiting
Majadahonda, Madrid, Spain
Hospital Universitario Virgen de La ArrixacaRecruiting
El Palmar, Murcia, Spain
Hospital Universitario Central de AsturiasRecruiting
Oviedo, Principality of Asturias, Spain
Hospital Universitario de BurgosRecruiting
Burgos, Spain
Local Institution - 0102Withdrawn
Madrid, Spain
Hospital Clinico San CarlosRecruiting
Madrid, Spain
Hospital Clinico Universitario Virgen de La VictoriaRecruiting
Málaga, Spain
Local Institution - 0113Not Yet Recruiting
London, Greater London, United Kingdom
Imperial College Healthcare NHS TrustRecruiting
London, Greater London, United Kingdom
University Of ManchesterRecruiting
Manchester, Greater Manchester, United Kingdom
Liverpool Heart and Chest HospitalRecruiting
Liverpool, Merseyside, United Kingdom
University Hospitals Birmingham NHS Foundation TrustRecruiting
Birmingham, West Midlands, United Kingdom
Leeds General InfirmaryRecruiting
Leeds, West Yorkshire, United Kingdom
University Hospital of WalesRecruiting
Cardiff, United Kingdom
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) · TrialPath