Mavacamten for Hypertrophic Cardiomyopathy

Not currently recruiting at 166 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: MyoKardia, Inc.

Must not be taking: Disopyramide, Ranolazine

Disqualifiers: ECG abnormalities, Syncope, 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: Long-term

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) for individuals with hypertrophic cardiomyopathy, a condition where the heart muscle thickens abnormally, potentially hindering blood pumping. Researchers aim to assess the effectiveness and safety of Mavacamten by testing different doses and dosing methods across three groups. Individuals who completed a related previous study and maintain a specific heart function level might be suitable for this trial. Participants should have no recent history of serious heart rhythm issues or other significant health conditions that could interfere with the study. As a Phase 2, Phase 3 trial, this research evaluates the treatment's effectiveness in an initial group and represents the final step before FDA approval, offering participants a chance to contribute to advancing treatment options.

Will I have to stop taking my current medications?

The trial requires that you stop taking disopyramide or ranolazine at least 14 days before the screening. Other medications are not specifically mentioned, so it's best to discuss your current medications with the study team.

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

Research has shown that mavacamten is generally safe and well-tolerated. In one study with 2,440 participants, less than 1% experienced a specific type of heart failure, indicating that serious heart-related side effects are rare.

Additionally, real-world data from different countries also demonstrated good results with mavacamten, supporting its safety for various groups. While no treatment is completely without risk, these findings suggest that mavacamten is safe for many individuals. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard of care for hypertrophic cardiomyopathy, which usually includes beta-blockers or calcium channel blockers, Mavacamten offers a novel approach by specifically targeting cardiac myosin. This unique mechanism of action directly addresses the heart muscle's contractility, potentially reducing the excessive thickening of the heart walls, which is a hallmark of the condition. Researchers are excited about Mavacamten because it may provide a more targeted and effective treatment option, with the possibility of improving symptoms and quality of life for patients in ways that current treatments might not achieve.

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

Research has shown that mavacamten can help reduce symptoms of hypertrophic cardiomyopathy (HCM), a condition where the heart muscle becomes too thick. Clinical studies have found that mavacamten lessens the blockage in the heart's main pumping chamber, a significant issue for people with HCM. One study showed that 43% of patients experienced improved heart function, as measured by improvements in their NYHA class symptoms, which indicate the severity of heart failure. Additionally, a review of multiple studies supports its effectiveness specifically for HCM, providing confidence in its use. These findings suggest that mavacamten could play an important role in managing this heart condition. Participants in this trial will receive mavacamten in different treatment arms, with varying target trough concentrations and dose titrations to assess its effectiveness. ^{1 3 6 7 8}

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 over 45 kg who completed the MAVERICK-HCM or EXPLORER-HCM studies, have a left ventricular ejection fraction (LVEF) of ≥50%, and can undergo accurate heart scans. Women must not be pregnant, breastfeeding, and use effective birth control if sexually active. Exclusions include certain ECG abnormalities, recent serious cardiac events or arrhythmias, current treatment with specific drugs like disopyramide or ranolazine, significant other diseases that could affect study results or compliance.

Inclusion Criteria

I weigh more than 45 kg.

Your blood and urine test results are within the normal range according to the lab's standards.

I am not pregnant or breastfeeding and will use effective birth control during and 90 days after the trial.

Exclusion Criteria

Is unable to comply with the study requirements, including the number of required visits to the clinical site.

Has any acute or serious comorbid condition (eg. major infection or hematologic, renal, metabolic, gastrointestinal, or endocrine dysfunction) that, in the judgment of the Investigator, could lead to premature termination of study participation or interfere with the measurement or interpretation of the efficacy and safety assessments in the study.

I have developed a significant cancer since joining the initial study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive active treatment with Mavacamten, with dosing adjusted based on target trough concentration or clinical response

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive Mavacamten to assess long-term safety

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Mavacamten

Trial Overview The study tests the long-term safety of Mavacamten in patients with Hypertrophic Cardiomyopathy who previously participated in related trials. It will take place across approximately 90 sites globally including some locations from previous studies to ensure continuity and reliability of data.

How Is the Trial Designed?

Group I: Group 3 Experimental Treatment 1 Intervention

Active Treatment for participants dose titrated to clinical response

Group II: Group 2 Experimental Treatment 1 Intervention

Active Treatment for participants with higher target trough concentration

Group III: Group 1 Experimental Treatment 1 Intervention

Active Treatment for participants with base target trough concentration

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?

MyoKardia, Inc.

Lead Sponsor

Trials

13

Recruited

1,000+

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, 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 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 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 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 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 (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 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]

Citations

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

CAMZYOS® (mavacamten) oHCM Clinical Study Results Amongst the efficacy outcomes, a range of 90 to 95 patients to date (of 231) have reached Week 180 for evaluation.

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 ... The analysis showed that Camzyos (mavacamten) was associated with reductions in left ventricular outflow tract (LVOT) obstruction and ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11522755/

A Systematic Review and Meta-analysis of Efficacy and ... Mavacamten, a first-in-class cardiac myosin inhibitor, is considered to be a specific drug for the treatment of HCM. This meta-analysis aimed to ...

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

Safety and Efficacy of Mavacamten and Aficamten in ... In MAVERICK‐HCM, following 16 weeks of treatment, mavacamten improved NYHA class ≥1 in 43% of patients compared with 37% for placebo (P=0.68), ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT03470545

Clinical Study to Evaluate Mavacamten (MYK-461) in ... This is a multicenter, international, double-blind study of the administration of mavacamten in participants with symptomatic obstructive HCM (oHCM).

6. 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.

7. medrxiv.org medrxiv.org/content/10.1101/2025.03.24.25324576v1.full-text

Characteristics and Outcomes of Mavacamten Use In 2440 ... The Revolution of Cardiac Myosin Inhibitors in Patients With Hypertrophic Cardiomyopathy. Can J Cardiol . 2024;40(5):800–819.

8. mayoclinicproceedings.org mayoclinicproceedings.org/article/S0025-6196(25)00029-1/abstract

Clinical Outcomes With Mavacamten Use in Patients ... The event rate of heart failure with reduced ejection fraction (HFrEF) remained low at less than 1% in both groups. Conclusion. In this ...