Mavacamten for Hypertrophic Cardiomyopathy

Not currently recruiting at 105 trial locations

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

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Bristol-Myers Squibb

Disqualifiers: Phenocopy diseases, LVEF <50%, others

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

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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: 200 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the effectiveness and safety of mavacamten, a medication for individuals with symptomatic obstructive hypertrophic cardiomyopathy (HCM), a condition where the heart muscle thickens, hindering blood flow. Researchers aim to evaluate how well mavacamten works and how the body processes it over time. Participants will receive either mavacamten or a placebo (a harmless pill resembling the real medicine) to compare results. This trial may suit those diagnosed with HCM who experience symptoms like shortness of breath or chest pain. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it mentions that you should not have any planned increases in your HCM medication dose. It's best to discuss your current medications with the trial team.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that mavacamten is generally well-tolerated, but important safety considerations exist. This treatment, already approved in the U.S. for adults with certain heart conditions, has been linked to serious side effects. One major risk is heart failure, where the heart can't pump blood effectively. Mavacamten can also reduce the heart's ability to push out blood, potentially leading to heart failure. These findings suggest that while mavacamten can help manage heart conditions, careful monitoring is essential to ensure safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Mavacamten is unique because it directly targets the underlying cause of hypertrophic cardiomyopathy (HCM) by modulating cardiac myosin, the protein responsible for heart muscle contraction. Unlike standard treatments like beta-blockers and calcium channel blockers that manage symptoms, mavacamten addresses the excessive thickening of the heart muscle itself. Researchers are excited about mavacamten because it offers a more precise approach to managing HCM, potentially improving heart function and quality of life for patients.

What evidence suggests that mavacamten might be an effective treatment for hypertrophic cardiomyopathy?

Research has shown that mavacamten may help treat symptoms of obstructive hypertrophic cardiomyopathy (HCM), a heart condition. One study found that patients taking mavacamten experienced better heart function and less blockage. Another study confirmed its benefits, showing that patients could exercise more easily and had fewer symptoms. Real-world evidence supports these findings, indicating that mavacamten can improve the quality of life for people with this condition. Overall, these results suggest that mavacamten could be a helpful treatment for managing HCM symptoms. Participants in this trial will receive either mavacamten or a placebo to further evaluate its effectiveness. ^{1 3 4 5 6}

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 adolescents with obstructive hypertrophic cardiomyopathy (HCM) who show symptoms. Specific details about eligibility criteria are not provided, but typically participants must meet certain health standards and may be required to have a specific level of disease severity.

Inclusion Criteria

I have been diagnosed with hypertrophic cardiomyopathy (HCM).

My heart has a blockage in the outflow tract.

I am experiencing symptoms.

Exclusion Criteria

Evidence of LVEF <50% in prior 6 months

My heart muscle thickening is not due to sarcomere dysfunction.

I am scheduled for a major heart procedure or an increase in my heart condition medication.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive mavacamten or placebo from day 1 to end of treatment at week 200

200 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4-8 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Mavacamten

Trial Overview The study tests the effectiveness and safety of a drug called Mavacamten compared to a placebo in young patients with HCM. It also looks at how the body processes the drug.

How Is the Trial Designed?

Group I: Placebo Experimental Treatment 1 Intervention

Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200.

Group II: Mavacamten Experimental Treatment 1 Intervention

Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200.

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 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 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]

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]

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 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 (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, 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 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]

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. 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 ...

4. 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.

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

EXPLORER-HCM: A Pivotal Trial for the Efficacy ... Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy ... heart failure or loss of effectiveness. Advise patients of the ...

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 CAMZYOS® (mavacamten) is the first and only cardiac myosin inhibitor approved in the U.S., indicated for the treatment of adults with symptomatic New York Heart ...