Clinical trial NCT03496168: Extension Study of Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Previously Enrolled in PIONEER
Official title: An Open-Label Extension Study of Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Previously Enrolled in Study MYK-461-004 (PIONEER)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hypertrophic Cardiomyopathy
Keywords: Symptomatic
Obstructive
Left ventricular outflow tract gradient
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2018-04-12
Enrollment: 13
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events and Treatment Emergent Serious Adverse Events
timeFrame: From first dose to end of treatment + 56 days (Approximately an average of 240 Weeks)
measure: Number of Participants Who Had Cardiovascular Death
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants Who Experienced Sudden Death
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants Who Were Hospitalized for Cardiovascular Reasons.
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants With Heart Failure Due to Systolic Dysfunction, Defined as Asymptomatic LVEF < 50%
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants With LVEF < 50% as Measured by Echocardiography.
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants Who Were Experienced Myocardial Infarction
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants With Ventricular Arrhythmias.
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants Who Experienced Syncope
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants Who Experienced Seizures
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants Who Were Experienced Strokes
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Number of Participants With a Change in QT and QTcF Intervals.
timeFrame: From first dose to end of study, (approximately 260 weeks)
measure: Post-exercise Left Ventricular Outflow Tract (LVOT) Gradient Over Time
timeFrame: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
measure: Resting Left Ventricular Outflow Tract (LVOT) Gradient Over Time
timeFrame: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
measure: Post Valsalva Left Ventricular Outflow Tract (LVOT) Gradient Over Time
timeFrame: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
measure: Participants With >= 1 NYHA Function Class Improvement
timeFrame: At Baseline, Week 4, Week 8, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144, Week 156, Week 180, Week 204, Week 228 and Week 252
measure: Mean Change From Baseline in the Overall KCCQ PRO Score.
timeFrame: At Baseline, Week 4, Week 8, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144, Week 156, Week 180, Week 204, Week 228 and Week 252
measure: Mean Change From Baseline in Serum NT-proBNP.
timeFrame: At Baseline, Week 4, Week 8, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144, Week 156, Week 180, Week 204, Week 228 and Week 252
measure: Number of Participants Who Received Septal Reduction Therapy
timeFrame: 252 weeks
measure: Plasma Concentration of Mavacamen Overtime
timeFrame: At Baseline, Week 4, Week 8, Week 24, Week 48, Week 72, Week 96, Week 120, Week 144, Week 156, Week 180, Week 204, Week 228 and Week 252
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Local Institution - 0003
state: Arizona
city: New Haven
country: United States
facility: Local Institution - 0001
state: Connecticut
city: Durham
country: United States
facility: Local Institution - 0004
state: North Carolina
city: Portland
country: United States
facility: Local Institution - 0002
state: Oregon
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: