Mavacamten for Hypertrophic Cardiomyopathy

(DISCOVER-HCM Trial)

Recruiting at 151 trial locations

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

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: Bristol-Myers Squibb

Must be taking: Beta blockers, non-DHP CCBs

Must not be taking: Investigational agents, cardiac myosin inhibitors

Disqualifiers: Phenocopy disease, LV hypertrophy, others

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

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: 18 months (European participants) or 5 years (United States participants)

1600 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness and safety of the drug mavacamten (also known as Camzyos or MYK-461) for individuals with symptomatic obstructive hypertrophic cardiomyopathy (HCM), a condition that thickens heart walls and impairs blood pumping. The study is open to participants in the U.S. and Europe who have this condition and are either taking mavacamten, using other heart medications, or not receiving treatment due to past issues or ineffectiveness. Suitable participants should experience noticeable symptoms like breathlessness or chest pain during activity and should either be on certain heart medications or have tried them previously.

As a Phase 3 trial, this study represents the final step before FDA approval, allowing participants to contribute to the potential availability of a new treatment.

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

The trial does not specify that you need to stop taking your current medications. Participants can continue with their existing treatments like beta blockers, calcium channel blockers, or disopyramide, or they can start mavacamten as part of the study.

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

Research has shown that mavacamten is generally safe for individuals with obstructive hypertrophic cardiomyopathy (oHCM). Studies have found no increased risk of negative or serious side effects. In fact, participants taking mavacamten reported side effects similar to those on other common treatments. Long-term research, up to 3.5 years, also supports its safety, showing consistent results over time. Based on available research, mavacamten appears to be well-tolerated by patients. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about Mavacamten for treating symptomatic obstructive hypertrophic cardiomyopathy (oHCM) because it offers a novel approach compared to traditional medications like beta-blockers, calcium channel blockers, and disopyramide. Mavacamten works by directly targeting the underlying cause of oHCM, which is the excessive contraction of heart muscle fibers. It does this by inhibiting cardiac myosin, which essentially helps the heart muscle relax more effectively. This mechanism is different from existing treatments, which primarily focus on symptom management rather than addressing the root of the problem.

What evidence suggests that this trial's treatments could be effective for obstructive hypertrophic cardiomyopathy?

Research has shown that mavacamten, one of the treatments in this trial, effectively treats symptomatic obstructive hypertrophic cardiomyopathy (HCM). Studies have found that mavacamten can reduce blockage in the heart's main pumping chamber, a major issue in HCM. This reduction can ease symptoms like shortness of breath and chest pain. In earlier studies, patients taking mavacamten experienced better heart health compared to those on standard treatments, including improvements in heart function and overall well-being. These findings suggest that mavacamten could be a promising treatment option for people with this heart condition. Participants in this trial may receive mavacamten or a standard treatment such as a beta-blocker, non-dihydropyridine calcium channel blocker, or disopyramide. ^{1 4 5 6 7}

Who Is on the Research Team?

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Are You a Good Fit for This Trial?

Inclusion Criteria

I have been diagnosed with obstructive hypertrophic cardiomyopathy.

I have been diagnosed with obstructive hypertrophic cardiomyopathy.

I can and will sign the consent form before starting the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive mavacamten or other standard care treatments for symptomatic obstructive hypertrophic cardiomyopathy

18 months (European participants) or 5 years (United States participants)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Mavacamten
• Other Treatments

How Is the Trial Designed?

Group I: Mavacamten Experimental Treatment 1 Intervention

Participants will receive mavacamten as prescribed by a physician according to standard of care for symptomatic oHCM

Group II: Beta-blocker (BB) / non-dihydropyridine (non-DHP) calcium channel blocker (CCB) / disopyramide Experimental Treatment 1 Intervention

Participants will receive BB/non-DHP CCB/disopyramide as prescribed by a physician according to standard of care for symptomatic oHCM

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

Citations

1.

camzyos.com

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

CAMZYOS® (mavacamten) oHCM Clinical Study Results

Clinical Study Results · What the data showed: · *Amongst the efficacy outcomes, a range of 90 to 95 patients to date (of 231) have reached Week 180 for ...

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.

mayoclinicproceedings.org

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

Clinical Outcomes With Mavacamten Use in Patients ...

Objective. To examine the association of mavacamten therapy with cardiovascular outcomes compared with standard medical management in patients ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07168655

Mavacamten in Obstructive Hypertrophic Cardiomyopathy

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment.

5.

ahajournals.org

ahajournals.org/doi/10.1161/JAHA.124.038758

Safety and Efficacy of Mavacamten and Aficamten in ...

In this review, we evaluated the pharmacologic profile and clinical outcomes for mavacamten and aficamten, 2 cardiac myosin inhibitors investigated in ...

6.

pmc.ncbi.nlm.nih.gov

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

A Systematic Review and Meta-analysis of Efficacy and ...

Mavacamten has great efficacy for the treatment of HCM. Meanwhile, mavacamten did not increase the incidence of adverse events or serious adverse events.

7.

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 ...

Cumulative analysis of data up to 3.5 years from EXPLORER-LTE showed consistent and sustained improvements in echocardiographic measures and ...