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

Stopped early · Phase 2

Conditions studied: Cardiomyopathy, Hypertrophic

In brief

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)

Key facts

Study ID

NCT05556343

Run by

Bristol-Myers Squibb

People needed

18

Starts

2023-01-18

Expected to finish

2025-02-27

Last updated by the study team

2026-06-02

Who can join

Age: 18 and older, up to 80. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Presence of any medical condition that precludes exercise stress testing.
• History of syncope or sustained ventricular tachyarrhythmia within 6 months prior to screening.
• Known infiltrative or storage disorder causing cardiac hypertrophy that mimics HCM, such as Fabry disease, amyloidosis, or Noonan syndrome with left ventricular hypertrophy.
• Prior treatment with mavacamten or aficamten. An exception may be made in cases where myosin inhibitor use was not within 4 months of the Screening visit, and with the agreement of both the Investigator and the Medical Monitor.
• Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the study (Note: Individuals with an unsuccessful myectomy or percutaneous ASA procedure performed > 6 months prior to Screening may be enrolled if study eligibility criteria for LVOT gradient criteria are met).
• Implantable cardioverter-defibrillator (ICD) placement or pulse generator change within 2 months prior to screening or planned new ICD placement during the study (pulse generator changes, if needed during the study are allowed).
• Has a history of resuscitated sudden cardiac arrest (any time) or known history of appropriate implantable cardioverter-defibrillator (ICD discharge for life-threatening ventricular arrhythmia within 6 months prior to screening.
• Has paroxysmal, atrial fibrillation with atrial fibrillation present per the Investigator's evaluation of the participant's ECG at the time of Screening.
• Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening (Note: Participants with persistent or permanent atrial fibrillation who are anticoagulated and adequately rate-controlled are allowed).
• Has QT interval with Fridericia correction (QTcF) > 500 msec when QRS interval < 120 msec or QTcF > 520 msec when QRS ≥ 120 msec if participant has left bundle branch block or any other 12-lead ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II).
• Has known moderate or severe (per investigator's judgment) aortic valve stenosis at screening.
• History of LV systolic dysfunction (LVEF < 45%) at any time during their clinical course.
• Clinically significant pulmonary disease associated with exertional dyspnea.
• Has known significant unrevascularized obstructive coronary artery disease (>70% stenosis in one or more main epicardial coronary arteries) or history of myocardial infarction Note: participants with prior coronary artery bypass grafting (CABG) or percutaneous coronary interventions (PCIs) are allowed if the procedure was performed at least 12 weeks prior to screening
• Prior treatment with cardiotoxic agents such as anthracyclines (eg, doxorubicin) or similar
• Other protocol-defined criteria apply.

Where it is running

• Local Institution - 0026 — La Jolla, California, United States
• Local Institution - 0014 — Los Angeles, California, United States
• Local Institution - 0016 — San Francisco, California, United States
• Local Institution - 0001 — Kansas City, Kansas, United States
• Local Institution - 0032 — New York, New York, United States
• Local Institution - 0013 — Durham, North Carolina, United States
• Local Institution - 0031 — Cincinnati, Ohio, United States
• Local Institution - 0015 — Cleveland, Ohio, United States
• Local Institution - 0024 — Portland, Oregon, United States
• Local Institution - 0021 — Nashville, Tennessee, United States
• Local Institution - 0025 — San Antonio, Texas, United States
• Local Institution - 0006 — Salt Lake City, Utah, United States
• Local Institution - 0027 — Bologna, BO, Italy
• Local Institution - 0005 — Florence, FI, Italy
• Local Institution - 0029 — Milan, Italy
• Local Institution - 0011 — Katowice, Silesian Voivodeship, Poland
• Local Institution - 0030 — Wroclaw, Poland
• Local Institution - 0028 — Alicante, A, Spain
• Local Institution - 0022 — Granada, GR, Spain
• Local Institution - 0008 — Málaga, MA, Spain
• Local Institution - 0002 — El Palmar, MU, Spain
• Local Institution - 0003 — Valencia, V, Spain
• Local Institution - 0009 — A Coruña, Spain
• Local Institution - 0023 — Barcelona, Spain
• Local Institution - 0010 — Majadahonda, Spain

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.