Phase 2 N=13 Treatment

Extension Study of Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Previously Enrolled in PIONEER

Hypertrophic Cardiomyopathy

Bottom Line

View on ClinicalTrials.gov: NCT03496168 ↗

Enrolled (actual)

Serious AEs

38.5%

Results posted

Jan 2025

Primary outcome: Primary: Number of Participants With Treatment Emergent Adverse Events and Treatment Emergent Serious Adverse Events — 13; 5 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: mavacamten (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Bristol-Myers Squibb
Primary completion: Nov 2023

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment Emergent Adverse Events and Treatment Emergent Serious Adverse Events	13; 5	—
PRIMARY Number of Participants Who Had Cardiovascular Death	—	—
PRIMARY Number of Participants Who Experienced Sudden Death	—	—
PRIMARY Number of Participants Who Were Hospitalized for Cardiovascular Reasons.	1	—
PRIMARY Number of Participants With Heart Failure Due to Systolic Dysfunction, Defined as Asymptomatic LVEF < 50%	—	—
PRIMARY Number of Participants With LVEF < 50% as Measured by Echocardiography.	2	—
PRIMARY Number of Participants Who Were Experienced Myocardial Infarction	—	—
PRIMARY Number of Participants With Ventricular Arrhythmias.	1; 0; 0; 0	—
PRIMARY Number of Participants Who Experienced Syncope	2; 1	—
PRIMARY Number of Participants Who Experienced Seizures	—	—
PRIMARY Number of Participants Who Were Experienced Strokes	—	—
PRIMARY Number of Participants With a Change in QT and QTcF Intervals.	7; 1; 1; 2; 1	—
PRIMARY Post-exercise Left Ventricular Outflow Tract (LVOT) Gradient Over Time	0; 0; 13; 0; 3; 10	—
PRIMARY Resting Left Ventricular Outflow Tract (LVOT) Gradient Over Time	3; 2; 8; 8; 0; 5	—
PRIMARY Post Valsalva Left Ventricular Outflow Tract (LVOT) Gradient Over Time	0; 1; 11; 3; 2; 8	—
PRIMARY Participants With >= 1 NYHA Function Class Improvement	15.4; 46.2; 76.9; 75.0; 90.9; 83.3	—
PRIMARY Mean Change From Baseline in the Overall KCCQ PRO Score.	5.809; 12.304; 16.931; 16.345; 18.600; 16.162	—
PRIMARY Mean Change From Baseline in Serum NT-proBNP.	-1017.4; -1440.6; -1512.8; -1579.2; -1708.4; -1579.1	—
PRIMARY Number of Participants Who Received Septal Reduction Therapy	—	—
PRIMARY Plasma Concentration of Mavacamen Overtime	166.51; 334.65; 396.18; 400.15; 407.27; 422.82	—

Summary

This is a multicenter open-label study of the administration of mavacamten in participants with symptomatic obstructive HCM (oHCM) who previously participated in study MYK-461-004 (PIONEER-HCM).

Eligibility Criteria

Key Inclusion Criteria

Completed Study MYK-461-004. Prior participation in a non-interventional observational study is allowed.
Body weight > 45 kg at Screening
Has safety laboratory parameters (chemistry and hematology) within normal limits

Key Exclusion Criteria

Has QTcF > > 500 ms or any other ECG abnormality considered by the investigator to pose a risk to subject safety (eg, second degree atrioventricular block type II)
Since enrollment into Study MYK-461-004, has developed obstructive coronary artery disease (> 70% stenosis in one or more arteries) or known moderate or severe aortic valve stenosis
Since enrollment into Study MYK-461-004, has developed any acute or serious comorbid condition (eg, major infection or hematologic, renal, metabolic, gastrointestinal, or endocrine dysfunction) that, in the opinion of the investigator or medical monitor, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion
Since enrollment into Study MYK-461-004 has developed clinically significant malignant disease

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03496168). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.