MYK-224 for Hypertrophic Cardiomyopathy

(MERCUTIO Trial)

No longer recruiting at 44 trial locations

Overseen By First line of the email MUST contain the NCT# and Site #.

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Bristol-Myers Squibb

Must not be taking: Mavacamten, Aficamten, Anthracyclines, others

Disqualifiers: Syncope, Ventricular tachyarrhythmia, Atrial fibrillation, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the safety and effectiveness of a new treatment, MYK-224, for individuals with obstructive Hypertrophic Cardiomyopathy (oHCM), a condition where the heart muscle thickens, hindering blood flow. Participants will receive either MYK-224 alone or in combination with common heart medications like beta-blockers or calcium channel blockers. The trial targets those diagnosed with oHCM who experience symptoms such as chest pain or shortness of breath. Eligible participants may continue with an optional, open-label extension period. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that MYK-224 is likely to be safe for humans?

Research shows that MYK-224 is still undergoing safety testing in people with obstructive Hypertrophic Cardiomyopathy, a condition where the heart muscle thickens. The trial is in Phase 2, indicating that some early safety data exists, but more information is needed to understand how well people tolerate it.

MYK-224 has been studied before, but one trial stopped for business reasons, not due to safety concerns. This suggests no major safety issues have emerged so far. However, since this is still an early stage of research, not all possible side effects are known yet. Participants in the current study will help gather more information on the safety and tolerability of MYK-224. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Hypertrophic Cardiomyopathy?

Researchers are excited about MYK-224 for hypertrophic cardiomyopathy because it offers a novel approach to tackling this heart condition. Unlike standard treatments, which typically involve beta-blockers or calcium channel blockers, MYK-224 targets the underlying mechanisms that cause the heart muscle to thicken. This potentially means addressing the root cause rather than just managing symptoms. Additionally, MYK-224 can be combined with existing treatments, providing a versatile option that could enhance the effectiveness of current therapies.

What evidence suggests that MYK-224 might be an effective treatment for Hypertrophic Cardiomyopathy?

Research shows that MYK-224, a drug that slows certain heart muscle activities, may help treat hypertrophic cardiomyopathy (HCM), a condition where the heart muscle becomes too thick. Earlier studies found that MYK-224 reduces blockages in the heart, improving symptoms and heart function in people with obstructive hypertrophic cardiomyopathy (oHCM). The drug targets proteins that cause the heart muscle to thicken, potentially reducing the extra muscle. In this trial, participants in Cohort 1 will receive MYK-224 either as a monotherapy or with a beta-blocker. Participants in Cohort 2 will receive MYK-224 with either a calcium channel blocker or disopyramide, combined with a beta-blocker or calcium channel blocker. Early results suggest that MYK-224 could be effective alone or with these common treatments. Although research is ongoing, initial findings are promising for those with oHCM. ^{1 2 4 6 7}

Who Is on the Research Team?

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Are You a Good Fit for This Trial?

This trial is for men and women with obstructive Hypertrophic Cardiomyopathy (oHCM) who have symptoms, a thickened heart wall, and specific heart function criteria. They must be able to undergo exercise stress testing and not have had certain recent cardiac procedures or treatments.

Inclusion Criteria

I experience mild to moderate heart symptoms.

I have been diagnosed with obstructive hypertrophic cardiomyopathy according to current guidelines.

My heart screening showed a specific high pressure reading.

Exclusion Criteria

I have been treated with heart-damaging drugs like doxorubicin.

I have had or will have an ICD placed or changed recently.

I have had heart issues with reduced pumping efficiency.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive MYK-224 either as a monotherapy or in combination with standard-of-care medications

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

What Are the Treatments Tested in This Trial?

Interventions

• MYK-224

Trial Overview The study tests MYK-224's effects on people with oHCM. It aims to understand how safe it is, how well it works, what the body does to the drug (pharmacokinetics), and how the drug affects the body (pharmacodynamics).

How Is the Trial Designed?

Group I: Cohort 2 Experimental Treatment 1 Intervention

Participants will receive MYK-224 in combination with standard-of-care consisting of either a calcium channel blocker or disopyramide (which is given in combination with either a beta-blocker or calcium channel blocker). Participants who complete Cohort 2 will be eligible for an optional open label extension period

Group II: Cohort 1 Experimental Treatment 1 Intervention

Participants will receive MYK-224 either as a monotherapy or in combination with standard-of-care consisting of a beta-blocker. Participants who complete Cohort 1 will be eligible for an optional open label extension period

Who Is Running the Clinical Trial?

Bristol-Myers Squibb

Lead Sponsor

Trials

2,731

Recruited

4,127,000+

Headquarters

New York City, USA

Known For

Oncology & Cardiovascular

Top Products

Eliquis, Opdivo, Revlimid, Orencia

Christopher Boerner

Bristol-Myers Squibb

Chief Executive Officer since 2023

PhD in Business Administration from the Haas School of Business, University of California, Berkeley; BA in Economics and History from Washington University in St. Louis

Deepak L. Bhatt

Bristol-Myers Squibb

Chief Medical Officer since 2024

MD from Yale University; MSc in Clinical Epidemiology from the University of Pennsylvania

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12383798/

Cardiac Myosin Inhibitors in the Treatment of Hypertrophic ... Hypertrophic cardiomyopathy (HCM) is a prevalent and often underdiagnosed genetic cardiac disorder characterized by left ventricular hypertrophy ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05556343

NCT05556343 | A Study to Evaluate the Efficacy, Safety ... The purpose of this study is to characterize the safety, tolerability, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of MYK-224 in participants ...

3. clinicaltrials.cedars-sinai.edu clinicaltrials.cedars-sinai.edu/view/CV029009

MYK-224 in Symptomatic Hypertrophic Cardiomyopathy and ... The purpose of this study is to examine the safety and effectiveness of an investigational drug called MYK-224 in individuals with obstructive hypertrophic.

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10766000/

Pharmaco-structural therapies for hypertrophic cardiomyopathy In this review, we redefine HCM as a treatable condition, evaluate current strategies for therapeutic intervention, and discuss novel myosin inhibitors.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT05556343?term=AREA%5BBasicSearch%5D(Disopyramide)&rank=4

A Study to Evaluate the Efficacy, Safety, and Tolerability of ... Data collected at the beginning of a clinical study for all participants and for each arm or comparison group. These data include demographics, such as age, sex ...

6. bmsstudyconnect.com bmsstudyconnect.com/ca/en/clinical-trials/NCT05556343.html

A Study to Evaluate the Efficacy, Safety, and Tolerability of ... A study to evaluate the efficacy, safety, and tolerability of MYK-224 in participants with symptomatic obstructive hypertrophic cardiomyopathy.

7. sigma.larvol.com sigma.larvol.com/product.php?e1=826692&tab=newstrac

MYK-224 News While mavacamten has established efficacy, next-generation agents like aficamten may offer improved safety and versatility. Further long-term studies are needed ...