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)

Obstructive Hypertrophic Cardiomyopathy Clinical Trial

— DISCOVER-HCM  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05489705
• Other study ID #: CV027-012
• Status: Recruiting
• Start date: August 16, 2022
• Est. completion date: August 15, 2029

Study information

• Verified date: April 2024
• Source: Bristol-Myers Squibb
• Contact: BMS Study Connect Contact Center www.BMSStudyConnect.com
• Phone: 855-907-3286
• Email: Clinical.Trials[@]bms.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1500
• Est. completion date: August 15, 2029
• Est. primary completion date: August 15, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

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])

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Hypertrophic
• Hypertrophy
• Obstructive Hypertrophic Cardiomyopathy

Locations

Country	Name	City	State
Puerto Rico	Research & Cardiovascular Corp	Ponce
United States	Alaska Heart Institute	Anchorage	Alaska
United States	UC Denver, AMC	Aurora	Colorado
United States	MedStar Health Research Institute	Baltimore	Maryland
United States	University Of Maryland, Ihv	Baltimore	Maryland
United States	St. Luke's University Health Network	Bethlehem	Pennsylvania
United States	Harvard (Massachusetts General Hospital)	Boston	Massachusetts
United States	Harvard Medical School - Brigham and Women's Hospital (BWH)	Boston	Massachusetts
United States	Focus Clinical Research	Charlotte	North Carolina
United States	The Rector and Visitors of the Univ of Virginia	Charlottesville	Virginia
United States	University of Chicago Dept of Medicine	Chicago	Illinois
United States	Christ Hospital Health Network	Cincinnati	Ohio
United States	Cleveland Clinic	Cleveland	Ohio
United States	University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Bassett Medical Center	Cooperstown	New York
United States	Baylor Scott & White Research Institute	Dallas	Texas
United States	North Texas Cardiology Center	Dallas	Texas
United States	Duke University Health System	Durham	North Carolina
United States	Spectrum Health Medical Group	Grand Rapids	Michigan
United States	Prisma Health - Upstate	Greenville	South Carolina
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	Hartford HealthCare	Hartford	Connecticut
United States	University of Hawaii Cancer Center	Honolulu	Hawaii
United States	BI Research Center	Houston	Texas
United States	The Methodist Hospital, Methodist Cancer Cencer	Houston	Texas
United States	Franciscan Physician Network-Indiana Heart Physicians	Indianapolis	Indiana
United States	Indiana. University	Indianapolis	Indiana
United States	University of Iowa Hospitals & Clinics	Iowa City	Iowa
United States	Uf Health Jacksonville	Jacksonville	Florida
United States	St. Luke's Mid-America Heart Institute	Kansas City	Missouri
United States	University Of Kansas Medical Center & Medical Pavilion	Kansas City	Kansas
United States	UC San Diego School of Medicine	La Jolla	California
United States	Lancaster General Hospital	Lancaster	Pennsylvania
United States	UAMS	Little Rock	Arkansas
United States	Keck School of Medicine of USC-Usc	Los Angeles	California
United States	University Of Wisconsin - Madison	Madison	Wisconsin
United States	Northwell Health	Manhasset	New York
United States	Medical College of Wisconsin, Inc (PI Address)	Milwaukee	Wisconsin
United States	Minneapolis Heart Institute Foundation	Minneapolis	Minnesota
United States	WVU Hospitals	Morgantown	West Virginia
United States	Morristown Medical Center	Morristown	New Jersey
United States	Saint Thomas Health	Nashville	Tennessee
United States	Tristar Centennial Medical Ctr	Nashville	Tennessee
United States	Mount Sinai Hospital	New York	New York
United States	Mount Sinai West	New York	New York
United States	CHI Health Reseach Center	Omaha	Nebraska
United States	Stanford Health Care Hospital & Clinics	Palo Alto	California
United States	AHN Allegheny General Hospital	Pittsburgh	Pennsylvania
United States	University Of Pittsburgh	Pittsburgh	Pennsylvania
United States	Baylor Scott & White The Heart Hospital Plano	Plano	Texas
United States	VCU Medical Center	Richmond	Virginia
United States	Carilion Clinic; Virginia Tech-Carilion School of Medicine	Roanoke	Virginia
United States	Saint Francis Hospital	Roslyn	New York
United States	Washington University	Saint Louis	Missouri
United States	University Of California San Francisco Medical Center	San Francisco	California
United States	Virginia Mason Medical Center	Seattle	Washington
United States	Baylor Scott & White Medical Centre - Temple	Temple	Texas
United States	Pima Heart and Vascular	Tucson	Arizona
United States	Cardiology Associates Research, Llc	Tupelo	Mississippi
United States	Westchester Medical Center	Valhalla	New York
United States	MedStar Washington Hospital Center	Washington	District of Columbia
United States	Henry Ford Health System	West Bloomfield	Michigan
United States	Geisinger Health System	Wilkes-Barre	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Bristol-Myers Squibb

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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%	Up to 5 Years
Secondary	Occurrence of arrhythmia	Incidence rates of participants with arrhythmia (new onset)	Up to 5 Years
Secondary	Occurrence of Major Adverse Cardiovascular Events (MACE)	A composite endpoint consisting of non-fatal acute Myocardial Infarction (MI), stroke, hospitalization due to Heart Failure (HF) and Cardiovascular (CV) mortality	Up to 5 Years
Secondary	Occurrence of non-fatal acute Myocardial Infarction (MI)		Up to 5 Years
Secondary	Occurrence of Stroke		Up to 5 Years
Secondary	Occurrence of hospitalization due to heart failure		Up to 5 Years
Secondary	Occurrence of cardiovascular mortality		Up to 5 Years
Secondary	Occurrence of all-cause mortality		Up to 5 Years
Secondary	Evaluation of functional responses: New York Heart Association (NYHA) function class	Percentage of Participants Whose NYHA Class Changes	Up to 5 Years
Secondary	Evaluation of functional responses: Left Ventricular Outflow Tract (LVOT) gradient	Mean Change From Baseline in LVOT Gradients At Rest and Provoked	Baseline, 5 Years
Secondary	Evaluation of functional responses: LVEF	Mean Change from Baseline in LVEF	Baseline, 5 Years
Secondary	Evaluation of patient reported outcome measure: Kansas City Cardiomyopathy Questionnaire 23 (KCCQ-23)	The KCCQ-23 is a disease-specific health status instrument composed of 23 items that quantifies the domains of physical limitations, symptoms, self-efficacy, social limitation and quality of life from heart failure. Scores range from 0-100, in which higher scores reflect better health status.	Baseline, 5 Years
Secondary	Evaluation of patient reported outcome measure: EuroQol Five Dimensions Questionnaire (5-level) (EQ-5D-5L): Health Utility Index	The EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L health utility index and EQ Visual Analog Scale (VAS). EQ-5D-5L health utility index is comprised of 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each has 5 levels of perceived problems (1-no problem, 2-slight problems, 3-moderate problems, 4-severe problems, 5-extreme problems). Participants select an answer for each of the 5 dimensions that best matches his/her health "today". Responses are used to generate a weighted summary index, which ranges from 0 to 1.00. A higher score indicates better health and positive changes from baseline indicate improvement of health.	Baseline, 5 Years
Secondary	Evaluation of patient reported outcome measure: EQ-5D-5L: VAS	The EQ-5D-5L is a 2-part instrument for use as a measure of health outcome, designed for self-completion by respondents. It consists of EQ-5D-5L descriptive system and VAS.; The VAS self-rating records the participant's own assessment of his or her overall health status at the time of completion, on a vertical line VAS with scale of 0 (the worst health you can imagine) to 100 (the best health you can imagine). A higher score indicates better health and positive changes from baseline indicate improvement of health status.	Baseline, 5 Years
Secondary	Evaluation of biomarkers of response: NT-proBNP	Change From Baseline in Serum concentration of NT-proBNP	Baseline, 5 Years
Secondary	Evaluation of biomarkers of response: Cardiac troponin	Change From Baseline in Serum Concentration of Cardiac Troponins	Baseline, 5 Years

External Links

•   BMS Clinical Trial Information