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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) (DISCOVER-HCM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05489705
Recruitment Status : Recruiting
First Posted : August 5, 2022
Last Update Posted : February 28, 2023
Sponsor:
Information provided by (Responsible Party):
Bristol-Myers Squibb

Study Description
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Brief 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.

Condition or disease
Obstructive Hypertrophic Cardiomyopathy

Study Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 1500 participants
Observational Model: Cohort
Time Perspective: Prospective
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cardiomyopathy

Groups and Cohorts
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Group/Cohort
Mavacamten
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


Outcome Measures
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Primary Outcome Measures :
  1. 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%


Secondary Outcome Measures :
  1. Occurrence of arrhythmia [ Time Frame: Up to 5 Years ]
    Incidence rates of participants with arrhythmia (new onset)

  2. 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

  3. Occurrence of non-fatal acute Myocardial Infarction (MI) [ Time Frame: Up to 5 Years ]
  4. Occurrence of Stroke [ Time Frame: Up to 5 Years ]
  5. Occurrence of hospitalization due to heart failure [ Time Frame: Up to 5 Years ]
  6. Occurrence of cardiovascular mortality [ Time Frame: Up to 5 Years ]
  7. Occurrence of all-cause mortality [ Time Frame: Up to 5 Years ]
  8. Evaluation of functional responses: New York Heart Association (NYHA) function class [ Time Frame: Up to 5 Years ]
    Percentage of Participants Whose NYHA Class Changes

  9. 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

  10. Evaluation of functional responses: LVEF [ Time Frame: Baseline, 5 Years ]
    Mean Change from Baseline in LVEF

  11. 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.

  12. 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.

  13. 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.


  14. Evaluation of biomarkers of response: NT-proBNP [ Time Frame: Baseline, 5 Years ]
    Change From Baseline in Serum concentration of NT-proBNP

  15. Evaluation of biomarkers of response: Cardiac troponin [ Time Frame: Baseline, 5 Years ]
    Change From Baseline in Serum Concentration of Cardiac Troponins


Eligibility Criteria
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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
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.
Criteria

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])
Contacts and Locations
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Information from the National Library of Medicine

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


Contacts
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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 #.

Locations
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Sponsors and Collaborators
Bristol-Myers Squibb
Investigators
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Study Director: Bristol-Myers Squibb Bristol-Myers Squibb
More Information
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Additional Information:

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Responsible Party: Bristol-Myers Squibb
ClinicalTrials.gov Identifier: NCT05489705    
Other Study ID Numbers: CV027-012
First Posted: August 5, 2022    Key Record Dates
Last Update Posted: February 28, 2023
Last Verified: February 2023
Keywords provided by Bristol-Myers Squibb:
Obstructive hypertrophic cardiomyopathy
Obstructive HCM (oHCM)
Mavacamten
Heart failure
oHCM
Additional relevant MeSH terms:
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Cardiomyopathies
Cardiomyopathy, Hypertrophic
Hypertrophy
Heart Diseases
Cardiovascular Diseases
 Pathological Conditions, Anatomical
Aortic Stenosis, Subvalvular
Aortic Valve Stenosis
Aortic Valve Disease
Heart Valve Diseases