Mavacamten for Hypertrophic Cardiomyopathy
Recruiting in Palo Alto (17 mi)
+98 other locations
Age: < 18
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Bristol-Myers Squibb
No Placebo Group
Pivotal Trial (Near Approval)
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 efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Do I have to stop taking my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you have a planned increase in your HCM medication dose, you may not be eligible.
Is the drug Mavacamten a promising treatment for Hypertrophic Cardiomyopathy?
Yes, Mavacamten is a promising drug for treating Hypertrophic Cardiomyopathy. It works by targeting the heart's muscle function to improve heart structure and function, helping to relieve symptoms and improve the quality of life for patients. It has been approved for use in the USA and offers a new option beyond traditional treatments.
13478What safety data is available for Mavacamten in treating hypertrophic cardiomyopathy?
Mavacamten, also known as Camzyos or MYK-461, has been evaluated in several clinical trials for its safety and efficacy in treating hypertrophic cardiomyopathy (HCM). Three randomized controlled trials (RCTs) involving 422 participants with a mean follow-up of 24 weeks have been conducted. These studies showed that Mavacamten was well tolerated, with improvements in exercise capacity and symptoms, and reduced the need for septal reduction therapy. The trials reported on serious adverse events (SAEs) and treatment-emergent adverse events (TEAEs), but specific safety data details can be found in the FDA approval documentation. Overall, Mavacamten has shown a favorable safety profile in the trials conducted so far.
35689What data supports the idea that Mavacamten for Hypertrophic Cardiomyopathy is an effective drug?
The available research shows that Mavacamten is effective for treating Hypertrophic Cardiomyopathy. In clinical trials, it was well tolerated and improved symptoms and exercise capacity. It also reduced the need for additional procedures by improving heart function. Compared to a placebo, Mavacamten led to better outcomes in patients, such as increased exercise ability and improved heart function, as shown in multiple studies.
24589Eligibility Criteria
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.
Exclusion Criteria
My heart muscle thickening is not due to sarcomere dysfunction.
Participant Groups
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.
2Treatment groups
Experimental Treatment
Group I: PlaceboExperimental Treatment1 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: MavacamtenExperimental Treatment1 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, Australia, 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 Australia 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)
Find A Clinic Near You
Research locations nearbySelect from list below to view details:
Local Institution - 0039Durham, NC
UPMC Childrens Hospital of PittsburghPittsburgh, PA
Stollery Children's HospitalEdmonton, Canada
Local Institution - 0009Indianapolis, IN
More Trial Locations
Loading ...
Who is running the clinical trial?
Bristol-Myers SquibbLead Sponsor
References
Mavacamten: a novel small molecule modulator of β-cardiac myosin for treatment of hypertrophic cardiomyopathy. [2022]Hypertrophic cardiomyopathy (HCM) is a common known monogenetic cardiovascular disorder which frequently leads to symptoms such as dyspnea and exercise intolerance. Current guideline-recommended pharmacotherapies have variable therapeutic responses. Mavacamten, a small molecule modulator of β-cardiac myosin, reduces hypercontractility, a central mechanism in the pathogenesis of HCM. Mavacamten has recently been evaluated in Phase 2 and 3 clinic trials for obstructive and nonobstructive symptomatic HCM.
Effect of Mavacamten on Echocardiographic Features in Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy. [2022]EXPLORER-HCM (Clinical Study to Evaluate Mavacamten [MYK-461] in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy) demonstrated that mavacamten, a cardiac myosin inhibitor, improves symptoms, exercise capacity, and left ventricular outflow tract (LVOT) obstruction in patients with obstructive hypertrophic cardiomyopathy (oHCM).
Mavacamten: First Approval. [2022]Mavacamten (Camzyos™) is an oral small-molecule cardiac myosin inhibitor developed by MyoKardia, Inc., a wholly owned subsidiary of Bristol Myers Squibb, for the treatment of hypertrophic cardiomyopathy (HCM) and diseases of diastolic dysfunction. In April 2022, mavacamten was approved for use in the USA in the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive HCM to improve functional capacity and symptoms. This article summarizes the milestones in the development of mavacamten leading to this first approval for the treatment of adults with symptomatic NYHA class II-III obstructive HCM.
Mavacamten, a First-in-Class Cardiac Myosin Inhibitor for Obstructive Hypertrophic Cardiomyopathy. [2023]To assess mavacamten's role in hypertrophic cardiomyopathy treatment.
Mavacamten Treatment for Hypertrophic Cardiomyopathy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials. [2022]Hypertrophic cardiomyopathy (HCM) is the most common heritable cardiomyopathy, yet pharmacological therapy has been unchanged for decades until the recent introduction of mavacamten, a first-in-class cardiac myosin inhibitor. We assessed the efficacy and safety of mavacamten in HCM. To date, only 3 randomized controlled trials (RCTs) compared the outcomes of mavacamten vs placebo for HCM. We used a fixed effects model to calculate risk ratios (RRs) with 95% confidence intervals (CIs). The primary composite endpoint (PCE) was defined as either ≥1.5 mL/kg/min increase in peak oxygen consumption (pVO2) with ≥1 New York Heart Association functional class (NYHA-FC) improvement or ≥3.0 mL/kg/min increase in pVO2 without worsening of NYHA-FC. Secondary outcomes included ≥1 NYHA-FC improvement, septal reduction therapy (SRT) or guideline eligible for SRT, ≥1 serious adverse event (SAE), ≥1 treatment emergency adverse event (TEAE), atrial fibrillation (AF), and nonsustained ventricular tachycardia (NSVT). Three RCTs (n = 422, mean follow-up 24 weeks) were included. Compared to placebo, mavacamten achieved higher rates of PCE (RR 1.92; 95% CI 1.28-2.88; P = 0.002) and ≥1 NYHA-FC improvement (RR 2.10; 95% CI 1.66-2.67; P
Safety and efficacy of mavacamten for treatment of hypertrophic cardiomyopathy: a systematic review and meta-analysis of randomized clinical trials. [2023]Mavacamten, an allosteric myosin inhibitor, is considered to be a promising drug for the treatment of hypertrophic cardiomyopathy (HCM). This meta-analysis aimed to explore the safety and efficacy of mavacamten in HCM patients.
Mavacamten, an Alternative to Septal Reduction Therapy for Patients with Hypertrophic Cardiomyopathy. [2023]Hypertrophic cardiomyopathy (HCM) is a common heridetary cardiac disorder characterized by a wide range of symptoms. The pharmacological treatment of HCM is currently limited to beta blockers, non-dihydropyridine calcium channel blockers and disopyramide. Mavacamten is a novel cardiac myosin inhibitor, which was recently added to the limited pharmacological list of treatment options for HCM. This editorial elaborates on current evidence evaluating the use of mavacamten in patients with symptomatic obstructive HCM, comments on its current use and its expanded potential applications in the future.
Mavacamten, a precision medicine for hypertrophic cardiomyopathy: From a motor protein to patients. [2023]Hypertrophic cardiomyopathy (HCM) is a primary myocardial disorder characterized by left ventricular hypertrophy, hyperdynamic contraction, and impaired relaxation of the heart. These functional derangements arise directly from altered sarcomeric function due to either mutations in genes encoding sarcomere proteins, or other defects such as abnormal energetics. Current treatment options do not directly address this causal biology but focus on surgical and extra-sarcomeric (sarcolemmal) pharmacological symptomatic relief. Mavacamten (formerly known as MYK-461), is a small molecule designed to regulate cardiac function at the sarcomere level by selectively but reversibly inhibiting the enzymatic activity of myosin, the fundamental motor of the sarcomere. This review summarizes the mechanism and translational progress of mavacamten from proteins to patients, describing how the mechanism of action and pharmacological characteristics, involving both systolic and diastolic effects, can directly target pathophysiological derangements within the cardiac sarcomere to improve cardiac structure and function in HCM. Mavacamten was approved by the Food and Drug Administration in April 2022 for the treatment of obstructive HCM and now goes by the commercial name of Camzyos. Full information about the risks, limitations, and side effects can be found at www.accessdata.fda.gov/drugsatfda_docs/label/2022/214998s000lbl.pdf.
Mavacamten: a first-in-class myosin inhibitor for obstructive hypertrophic cardiomyopathy. [2023]Mavacamten is a first-in-class, targeted, cardiac-specific myosin inhibitor approved by the US Food and Drug Administration for the treatment of adults with symptomatic New York Heart Association Classes II and III obstructive hypertrophic cardiomyopathy (oHCM). Mavacamten was developed to target the hyper-contractile phenotype, which plays a critical role in the pathophysiology of the disease. In Phase 2 and 3 clinical trials, mavacamten was well tolerated, reduced left ventricular outflow tract gradients, improved exercise capacity and symptoms, and was associated with improvements in other clinically relevant parameters, such as patient-reported outcomes and circulating biomarkers. In addition, treatment with mavacamten was associated with evidence of favourable cardiac remodelling in multi-modality imaging studies. Mavacamten substantially reduced guideline eligibility for septal reduction therapy candidates with oHCM and drug-refractory symptoms. In this article, the available efficacy and safety data from completed and ongoing clinical studies of mavacamten in patients with symptomatic oHCM are reviewed. Longer term extension studies may help address questions related to the positioning of mavacamten in current oHCM management algorithms, interactions with background therapy, as well as the potential for disease modification beyond symptomatic relief of left ventricular outflow tract obstruction.