TerminatedPhase 2

A Study to Evaluate the Efficacy, Safety, and Tolerability of MYK-224 in Participants With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Stopped: Business objectives have changed

United States, Italy, Poland18 participantsStarted 2023-01-18

Plain-language summary

The purpose of this study is to characterize the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of MYK-224 in participants with obstructive Hypertrophic Cardiomyopathy (oHCM)

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Has adequate acoustic windows, to enable accurate TTEs as determined by the echocardiography core laboratory. * Men or women diagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines, satisfying both of the following criteria: * Has unexplained left ventricular (LV) hypertrophy with nondilated ventricular chambers in the absence of other cardiac (eg, hypertension, aortic stenosis) or systemic disease and with maximal LV wall thickness ≥ 15 millimeter (mm) (or ≥ 13 mm with positive family history of hypertrophic cardiomyopathy or with a known disease-causing mutation), as determined by core laboratory interpretation. AND \-- Has a LVOT peak gradient during screening as assessed by echocardiography of ≥ 50 millimeters of mercury (mm Hg) at rest, or ≥ 30 mm Hg at rest and ≥ 50 mm Hg with Valsalva maneuver (confirmed by echocardiography core laboratory interpretation). * Has resting LVEF ≥ 60% at the Screening visit as determined by echocardiography core laboratory. * New York Heart Association (NYHA) functional class II or III symptoms at screening. * Has a valid measurement of LVOT post-exercise peak gradient at screening as determined by echocardiography core laboratory. Exclusion Criteria: * Presence of any medical condition that precludes exercise stress testing. * History of syncope or sustained ventricular tachyarrhythmia within 6 months prior…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part A: Number of Participants With Adverse Events and Serious Adverse Events

Timeframe: From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Number of Participants With at Least One Event of Arrhythmias

Timeframe: From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Number of Participants With at Least One Event of Appropriate Implantable Cardioverter Defibrillator Therapy and Resuscitated Cardiac Arrest

Timeframe: From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Change From Baseline in Vital Signs - Heart Rate

Timeframe: Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Change From Baseline in Vital Signs - Mean Systolic Blood Pressure

Timeframe: Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Change From Baseline in Vital Signs - Mean Diastolic Blood Pressure

Trial details

NCT IDNCT05556343

SponsorBristol-Myers Squibb

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-08-19

Results submitted2026-02-27

View on ClinicalTrials.gov More Cardiomyopathy, Hypertrophic trials

Who can participate

Age range

18 Years – 80 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Part A: Number of Participants With Adverse Events and Serious Adverse Events

Timeframe: From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Number of Participants With at Least One Event of Arrhythmias

Timeframe: From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Number of Participants With at Least One Event of Appropriate Implantable Cardioverter Defibrillator Therapy and Resuscitated Cardiac Arrest

Timeframe: From first dose (Day 1) until study end date in Part A or 30 days after early termination date (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Change From Baseline in Vital Signs - Heart Rate

Timeframe: Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Change From Baseline in Vital Signs - Mean Systolic Blood Pressure

Timeframe: Baseline and until treatment end date in Part A (Median 17.55 weeks and Maximum 54.9 weeks)

Part A: Change From Baseline in Vital Signs - Mean Diastolic Blood Pressure