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) (DISCOVER-HCM)
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|ClinicalTrials.gov Identifier: NCT05489705|
Recruitment Status : Recruiting
First Posted : August 5, 2022
Last Update Posted : February 28, 2023
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|Condition or disease|
|Obstructive Hypertrophic Cardiomyopathy|
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||1500 participants|
|Target Follow-Up Duration:||5 Years|
|Official Title:||Deliver Insights in Hypertrophic Cardiomyopathy and Observational Outcomes in Real World: United States Prospective Registry Study|
|Actual Study Start Date :||August 16, 2022|
|Estimated Primary Completion Date :||August 15, 2029|
|Estimated Study Completion Date :||August 15, 2029|
Participants will receive mavacamten as prescribed by a physician according to standard of care for symptomatic oHCM
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
- 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 [ Time Frame: Up to 5 Years ]Systolic dysfunction defined as symptomatic Left Ventricular Ejection Fraction (LVEF) < 50%
- Occurrence of arrhythmia [ Time Frame: Up to 5 Years ]Incidence rates of participants with arrhythmia (new onset)
- Occurrence of Major Adverse Cardiovascular Events (MACE) [ Time Frame: Up to 5 Years ]A composite endpoint consisting of non-fatal acute Myocardial Infarction (MI), stroke, hospitalization due to Heart Failure (HF) and Cardiovascular (CV) mortality
- Occurrence of non-fatal acute Myocardial Infarction (MI) [ Time Frame: Up to 5 Years ]
- Occurrence of Stroke [ Time Frame: Up to 5 Years ]
- Occurrence of hospitalization due to heart failure [ Time Frame: Up to 5 Years ]
- Occurrence of cardiovascular mortality [ Time Frame: Up to 5 Years ]
- Occurrence of all-cause mortality [ Time Frame: Up to 5 Years ]
- Evaluation of functional responses: New York Heart Association (NYHA) function class [ Time Frame: Up to 5 Years ]Percentage of Participants Whose NYHA Class Changes
- Evaluation of functional responses: Left Ventricular Outflow Tract (LVOT) gradient [ Time Frame: Baseline, 5 Years ]Mean Change From Baseline in LVOT Gradients At Rest and Provoked
- Evaluation of functional responses: LVEF [ Time Frame: Baseline, 5 Years ]Mean Change from Baseline in LVEF
- Evaluation of patient reported outcome measure: Kansas City Cardiomyopathy Questionnaire 23 (KCCQ-23) [ Time Frame: Baseline, 5 Years ]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.
- Evaluation of patient reported outcome measure: EuroQol Five Dimensions Questionnaire (5-level) (EQ-5D-5L): Health Utility Index [ Time Frame: Baseline, 5 Years ]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.
- Evaluation of patient reported outcome measure: EQ-5D-5L: VAS [ Time Frame: Baseline, 5 Years ]
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.
- Evaluation of biomarkers of response: NT-proBNP [ Time Frame: Baseline, 5 Years ]Change From Baseline in Serum concentration of NT-proBNP
- Evaluation of biomarkers of response: Cardiac troponin [ Time Frame: Baseline, 5 Years ]Change From Baseline in Serum Concentration of Cardiac Troponins
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|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
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.
- Has documented LVEF of ≥ 55% recorded by echocardiography within the last 6 months.
- Symptoms consistent with NYHA functional class II-IV.
- ≥ 18 years of age at the time of informed consent.
- 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.
- Known phenocopy disease (eg, 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, non-DHP CCBs, or disopyramide).
- 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])
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05489705
|Contact: BMS Study Connect Contact Center www.BMSStudyConnect.com||855-907-3286||Clinical.Trials@bms.com|
|Contact: First line of the email MUST contain the NCT# and Site #.|
|Study Director:||Bristol-Myers Squibb||Bristol-Myers Squibb|
|Responsible Party:||Bristol-Myers Squibb|
|Other Study ID Numbers:||
|First Posted:||August 5, 2022 Key Record Dates|
|Last Update Posted:||February 28, 2023|
|Last Verified:||February 2023|
Obstructive hypertrophic cardiomyopathy
Obstructive HCM (oHCM)
Pathological Conditions, Anatomical
Aortic Stenosis, Subvalvular
Aortic Valve Stenosis
Aortic Valve Disease
Heart Valve Diseases