Mavacamten for Hypertrophic Cardiomyopathy

(MEMENTO Trial)

Recruiting at 43 trial locations

Overseen By BMS Study Connect Contact Center www.BMSStudyConnect.com

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 4

Recruiting

Sponsor: Bristol-Myers Squibb

Disqualifiers: Coronary artery disease, Cardiac arrest, others

No Placebo Group

Prior Safety Data

Breakthrough Therapy

Approved in 9 Jurisdictions

Trial Summary

What is the purpose of this trial?

The purpose of this study is to evaluate the mavacamten impact on myocardial structure with cardiac magnetic resonance imaging (CMR) in adult participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) \[New York Heart Association (NYHA) Functional Class II or III\].

Will I have to stop taking my current medications?

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

What data supports the effectiveness of the drug Mavacamten for treating hypertrophic cardiomyopathy?

Research shows that Mavacamten, a drug that helps the heart muscle work better, is effective for treating hypertrophic cardiomyopathy. It improves symptoms, exercise capacity, and heart function, and has been shown to be better than a placebo in clinical trials. ^{1 2 3 4 5}

Is Mavacamten safe for humans?

Mavacamten, also known as Camzyos or MYK-461, has been tested in clinical trials for hypertrophic cardiomyopathy and was generally well tolerated, with some patients experiencing serious adverse events. It was approved by the FDA in April 2022, indicating that its benefits outweigh the risks for this condition. ^{2 3 4 6 7}

How is the drug Mavacamten unique in treating hypertrophic cardiomyopathy?

Mavacamten is unique because it directly targets the heart's muscle fibers by inhibiting myosin, the protein responsible for muscle contraction, which helps reduce the excessive contraction seen in hypertrophic cardiomyopathy. Unlike other treatments that focus on symptom relief, Mavacamten addresses the underlying cause of the condition. ^{1 2 6 8 9}

Research Team

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Eligibility Criteria

This trial is for adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), specifically those who experience certain levels of heart blockage and have a normal or near-normal heart pumping function. They should be somewhat limited in their physical activity but not bedridden. People with recent severe cardiac events, pacemakers, conditions that look like oHCM, or major artery blockages can't join.

Inclusion Criteria

I have been diagnosed with a specific heart condition (oHCM) that meets certain criteria.

Exclusion Criteria

You have a heart device like a pacemaker or defibrillator, or another reason that makes it unsafe for you to have a magnetic resonance imaging (MRI) of your heart.

I have a condition that causes thickening of the heart muscle, similar to oHCM.

I had a severe heart rhythm problem or cardiac arrest in the last 6 months.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive mavacamten to evaluate its impact on myocardial structure with cardiac magnetic resonance imaging (CMR)

48 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Mavacamten (Myosin Inhibitor)

Trial Overview The study tests the effects of Mavacamten on the heart's structure using advanced imaging techniques in people with oHCM. Participants will either receive Mavacamten or a placebo to compare outcomes between the two groups.

Participant Groups

Group I: Mavacamten Experimental Treatment 1 Intervention

Mavacamten is already approved in Canada, Switzerland, Brazil for the following indications:

Approved in Canada as Camzyos for:

• Symptomatic obstructive hypertrophic cardiomyopathy (oHCM)

Approved in Switzerland as Camzyos for:

• Symptomatic obstructive hypertrophic cardiomyopathy (oHCM)

Approved in Brazil as Camzyos for:

• Symptomatic obstructive hypertrophic cardiomyopathy (oHCM)

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

Findings from Research

Mavacamten effectively reduces left ventricular outflow obstruction and improves heart function in patients with obstructive hypertrophic cardiomyopathy, offering a new treatment option for those who do not respond to standard therapies.

Despite its efficacy, mavacamten is not considered cost-effective, with a high acquisition cost leading to significant expenses for additional quality-adjusted life years, and its role in treating nonobstructive hypertrophic cardiomyopathy remains unclear.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavacamten, a First-in-Class Cardiac Myosin Inhibitor for Obstructive Hypertrophic Cardiomyopathy. Dalo, JD., Weisman, ND., White, CM.[2023]

Mavacamten (Camzyos) is a newly approved treatment for obstructive hypertrophic cardiomyopathy (HCM) that works by selectively inhibiting myosin, a key protein in the heart's muscle cells, to improve heart function directly at the sarcomere level.

This treatment addresses the underlying causes of HCM, unlike current options that mainly provide symptomatic relief, potentially leading to better outcomes for patients with this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavacamten, a precision medicine for hypertrophic cardiomyopathy: From a motor protein to patients. Nag, S., Gollapudi, SK., Del Rio, CL., et al.[2023]

Mavacamten, a new cardiac myosin inhibitor, significantly improves exercise capacity and functional status in patients with hypertrophic cardiomyopathy (HCM), achieving higher rates of primary composite endpoints compared to placebo in three randomized controlled trials involving 422 participants over a mean follow-up of 24 weeks.

While mavacamten reduces the need for septal reduction therapy (SRT), it is associated with higher rates of treatment-emergent adverse events (TEAEs), indicating a need for further research on its long-term safety and efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavacamten Treatment for Hypertrophic Cardiomyopathy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. Ismayl, M., Abbasi, MA., Marar, R., et al.[2022]

In a study of 251 patients with symptomatic obstructive hypertrophic cardiomyopathy, mavacamten significantly improved cardiac function, with 80.9% of patients showing complete resolution of mitral valve systolic anterior motion after 30 weeks, compared to only 34.0% in the placebo group.

Mavacamten also led to significant improvements in diastolic function, as evidenced by reductions in left atrial volume index and lateral E/e', which were associated with decreased levels of a biomarker indicating myocardial wall stress, highlighting its efficacy in addressing key pathophysiological aspects of the condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effect of Mavacamten on Echocardiographic Features in Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy. Hegde, SM., Lester, SJ., Solomon, SD., et al.[2022]

Mavacamten (Camzyos™) is an oral medication specifically designed to inhibit cardiac myosin, and it was approved in April 2022 for treating adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM) classified as NYHA class II-III.

The approval of mavacamten marks a significant milestone in HCM treatment, as it aims to improve both functional capacity and symptoms in affected patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavacamten: First Approval. Keam, SJ.[2022]

Mavacamten has been shown to be effective in improving clinical responses and reducing NYHA function class in patients with hypertrophic cardiomyopathy (HCM), based on a meta-analysis of 539 patients across four clinical trials.

The drug was well-tolerated with no significant increase in serious adverse events compared to placebo, indicating its safety for use in HCM patients, particularly those with hypertrophic obstructive cardiomyopathy (HOCM).

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials. Rabiee Rad, M., Ghasempour Dabaghi, G., Habibi, D.[2023]

Mavacamten is a new treatment option for hypertrophic cardiomyopathy (HCM), a hereditary heart condition, expanding the limited pharmacological options currently available, which include beta blockers and calcium channel blockers.

Current evidence suggests that mavacamten is particularly effective for patients with symptomatic obstructive HCM, indicating its potential to significantly improve patient outcomes in this specific group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavacamten, an Alternative to Septal Reduction Therapy for Patients with Hypertrophic Cardiomyopathy. Desai, MY., Hajj Ali, A.[2023]

Mavacamten has shown promising efficacy in treating symptomatic hypertrophic cardiomyopathy (HCM) in Phase 2 and 3 trials, leading to significant improvements in symptoms and physiological metrics, particularly in patients with obstructive HCM.

The treatment resulted in minimal adverse events, indicating a favorable safety profile, while effectively reducing the left ventricular outflow tract (LVOT) gradient and improving quality of life measures such as the Kansas City Cardiomyopathy Questionnaire (KCCQ) scores.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavacamten: a novel small molecule modulator of β-cardiac myosin for treatment of hypertrophic cardiomyopathy. Tower-Rader, A., Ramchand, J., Nissen, SE., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

Mavacamten, a First-in-Class Cardiac Myosin Inhibitor for Obstructive Hypertrophic Cardiomyopathy. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Mavacamten, a precision medicine for hypertrophic cardiomyopathy: From a motor protein to patients. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Mavacamten: a first-in-class myosin inhibitor for obstructive hypertrophic cardiomyopathy. [2023]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Mavacamten Treatment for Hypertrophic Cardiomyopathy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. [2022]

5. United States pubmed.ncbi.nlm.nih.gov

Effect of Mavacamten on Echocardiographic Features in Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy. [2022]

6. New Zealand pubmed.ncbi.nlm.nih.gov

Mavacamten: First Approval. [2022]

7. Germany pubmed.ncbi.nlm.nih.gov

Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials. [2023]

8. England pubmed.ncbi.nlm.nih.gov

Mavacamten, an Alternative to Septal Reduction Therapy for Patients with Hypertrophic Cardiomyopathy. [2023]

9. England pubmed.ncbi.nlm.nih.gov

Mavacamten: a novel small molecule modulator of β-cardiac myosin for treatment of hypertrophic cardiomyopathy. [2022]