Mavacamten for Hypertrophic Cardiomyopathy

(MEMENTO Trial)

Not currently recruiting at 56 trial locations

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

Age: 18+

Sex: Any

Trial Phase: Phase 4

Sponsor: Bristol-Myers Squibb

Disqualifiers: Coronary artery disease, Cardiac arrest, 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

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 9 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 48 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a drug called mavacamten (also known as Camzyos or MYK-461) to assess its effects on the heart's structure in people with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). In oHCM, the heart muscle thickens, hindering blood flow. The study uses a special heart scan to monitor changes in the heart. Suitable participants have been diagnosed with oHCM and experience noticeable symptoms, such as fatigue or shortness of breath during activities. As a Phase 4 trial, this research aims to understand how the already FDA-approved and effective treatment benefits more patients.

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 is the safety track record for Mavacamten?

Research has shown that mavacamten, a treatment for obstructive hypertrophic cardiomyopathy (a condition where the heart muscle thickens), is generally well-tolerated by patients. Studies have found that patients using mavacamten often experience symptom improvements. However, some risks exist. Mavacamten can cause serious side effects, including heart failure, which occurs when the heart cannot pump enough blood to meet the body's needs.

The FDA has already approved the treatment for certain patients with this heart condition, indicating that its safety has been thoroughly studied. Still, anyone considering joining a trial should discuss the potential risks and benefits with their healthcare provider. ^{1 2 3 4 5}

Why are researchers enthusiastic about this study treatment?

Mavacamten is unique because it directly targets the underlying cause of hypertrophic cardiomyopathy, rather than just managing symptoms like most current treatments. Unlike beta-blockers and calcium channel blockers, which focus on relieving symptoms, Mavacamten works by reducing excessive cardiac muscle contraction. This new mechanism of action offers the potential to improve heart function and symptoms more effectively, which is why researchers are excited about its potential benefits for patients.

What is the effectiveness track record for Mavacamten in treating hypertrophic cardiomyopathy?

Research has shown that mavacamten, the treatment under study in this trial, effectively treats obstructive hypertrophic cardiomyopathy (oHCM), a condition characterized by an overly thick heart muscle. Studies have found that mavacamten improves symptoms and heart function in people with oHCM by reducing excessive muscle contractions, a major issue in oHCM. Long-term data confirmed its safety and effectiveness, making it the first approved treatment of its kind for this condition. Real-world evidence supports its benefits, demonstrating positive results in various groups of people. ^{2 3 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 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.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Mavacamten

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.

How Is the Trial Designed?

Group I: Mavacamten Experimental Treatment 1 Intervention

Mavacamten is already approved in United States, European Union, Canada, Switzerland, Brazil for the following indications:

Approved in United States as Camzyos for:

• Symptomatic obstructive hypertrophic cardiomyopathy (oHCM)

Approved in European Union as Camzyos for:

• Symptomatic obstructive hypertrophic cardiomyopathy (oHCM)

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

Published Research Related to This Trial

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 is a targeted cardiac-specific myosin inhibitor approved for treating adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), showing significant efficacy in reducing left ventricular outflow tract gradients and improving exercise capacity in Phase 2 and 3 clinical trials.

The treatment was well tolerated and led to favorable cardiac remodeling, reducing the need for invasive septal reduction therapy in patients with drug-refractory symptoms, indicating its potential as a key option in managing oHCM.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mavacamten: a first-in-class myosin inhibitor for obstructive hypertrophic cardiomyopathy. Braunwald, E., Saberi, S., Abraham, TP., et al.[2023]

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, 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]

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 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]

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 (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]

Citations

1. camzyos.com camzyos.com/what-is-camzyos/study-results

CAMZYOS® (mavacamten) oHCM Clinical Study Results See how CAMZYOS® (mavacamten) may help symptomatic obstructive cardiomyopathy (oHCM) and see clinical study results and side effects ... hypertrophic ...

2. news.bms.com news.bms.com/news/corporate-financial/2025/Bristol-Myers-Squibb-Presents-Real-World-Outcomes-of-Camzyos-mavacamten-Across-Four-Continents-at-the-European-Society-of-Cardiology-ESC-Congress-2025/default.aspx

Findings from COLLIGO-HCM reinforce Camzyos' efficacy ... ... Hypertrophic CardioMyopathy) is Bristol Myers Squibb's global real-world data program. ... CAMZYOS® (mavacamten) is the first and only cardiac ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11301103/

Efficacy and safety of Mavacamten for symptomatic ... Hypertrophic cardiomyopathy (HCM) is an autosomal dominant disorder with risk of sudden cardiac death (SCD) in children and adolescents.

4. ahajournals.org ahajournals.org/doi/10.1161/JAHA.124.038758

Safety and Efficacy of Mavacamten and Aficamten in ... Cardiac myosin inhibitors were recently developed to address the underlying pathophysiology of hypertrophic cardiomyopathy and to improve ...

5. news.bms.com news.bms.com/news/details/2024/Long-Term-Follow-Up-Data-from-Phase-3-Study-of-CAMZYOS-mavacamten-Underscores-Established-Efficacy-and-Safety-Profile-in-Patients-with-Symptomatic-Obstructive-Hypertrophic-Cardiomyopathy-oHCM/default.aspx

Long-Term Follow-Up Data from Phase 3 Study of ... CAMZYOS is the first and only approved cardiac myosin inhibitor that targets the source of symptomatic obstructive hypertrophic cardiomyopathy.

6. news.bms.com news.bms.com/news/details/2025/Bristol-Myers-Squibb-Showcases-New-Long-Term-and-Real-World-Data-From-Cardiovascular-Portfolio-at-the-American-Heart-Association-Scientific-Sessions-2025/default.aspx

Corporate news details ... safety profile of Camzyos (mavacamten) in symptomatic obstructive hypertrophic cardiomyopathy ... Patient-reported outcomes in patients with symptomatic ...

7. camzyoshcp.com camzyoshcp.com/explorer-hcm/pivotal-trial

EXPLORER-HCM: A Pivotal Trial for the Efficacy ... IMPORTANT SAFETY INFORMATION · WARNING: RISK OF HEART FAILURE · CAMZYOS reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to ...