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

January 6, 2025 updated by: Bristol-Myers Squibb

An Open-Label Extension Study of Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy Previously Enrolled in Study MYK-461-004 (PIONEER)

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
13

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arizona
      • Scottsdale, Arizona, United States, 85259
        • Local Institution - 0003
    • Connecticut
      • New Haven, Connecticut, United States, 06520-8017
        • Local Institution - 0001
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Local Institution - 0004
    • Oregon
      • Portland, Oregon, United States, 97239
        • Local Institution - 0002

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: mavacamten (MYK-461)
Drug: mavacamten
mavacamten capsules
Other Names:
  • MYK-461

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants With Treatment Emergent Adverse Events and Treatment Emergent Serious Adverse Events
Time Frame: From first dose to end of treatment + 56 days (Approximately an average of 240 Weeks)

AE is defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product; it does not necessarily have to have a causal relationship with this treatment.

An SAE is defined as any untoward medical occurrence at any dose that:

  • Results in death
  • Is immediately life-threatening (places the participant at immediate risk of death from the event as it occurred)
  • Requires inpatient hospitalization or prolongation of existing hospitalization
  • Results in persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions
  • Results in a congenital abnormality or birth defect
  • Is an important medical event that may not result in death, be life- threatening, or require hospitalization, but may be considered an SAE when, based upon appropriate medical judgment, it may require medical or surgical intervention to prevent any of the outcomes listed above.
From first dose to end of treatment + 56 days (Approximately an average of 240 Weeks)
Number of Participants Who Had Cardiovascular Death
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants who had died due to cardiovascular reasons.
From first dose to end of study, (approximately 260 weeks)
Number of Participants Who Experienced Sudden Death
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants who experienced sudden death
From first dose to end of study, (approximately 260 weeks)
Number of Participants Who Were Hospitalized for Cardiovascular Reasons.
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants who were hospitalized for cardiovascular reasons.
From first dose to end of study, (approximately 260 weeks)
Number of Participants With Heart Failure Due to Systolic Dysfunction, Defined as Asymptomatic LVEF < 50%
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants with heart failure due to systolic dysfunction, defined as asymptomatic LVEF < 50%
From first dose to end of study, (approximately 260 weeks)
Number of Participants With LVEF < 50% as Measured by Echocardiography.
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants with LVEF < 50% as measured by echocardiography.
From first dose to end of study, (approximately 260 weeks)
Number of Participants Who Were Experienced Myocardial Infarction
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants who experienced myocardial infarction.
From first dose to end of study, (approximately 260 weeks)
Number of Participants With Ventricular Arrhythmias.
Time Frame: From first dose to end of study, (approximately 260 weeks)

Types of Ventricular Arrhythmias measured in this endpoint will be:

Ventricular Tachycardia Ventricular Fibrilation Ventricular Flutter
From first dose to end of study, (approximately 260 weeks)
Number of Participants Who Experienced Syncope
Time Frame: From first dose to end of study, (approximately 260 weeks)

Number of participants who experienced syncope.

Syncope will be defined as participants who experienced dizziness or orthostatic hypotension.
From first dose to end of study, (approximately 260 weeks)
Number of Participants Who Experienced Seizures
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants who were experienced seizures.
From first dose to end of study, (approximately 260 weeks)
Number of Participants Who Were Experienced Strokes
Time Frame: From first dose to end of study, (approximately 260 weeks)
Number of participants who were experienced strokes.
From first dose to end of study, (approximately 260 weeks)
Number of Participants With a Change in QT and QTcF Intervals.
Time Frame: From first dose to end of study, (approximately 260 weeks)

Number of participants with a change in QTcF intervals.

QTcF: An electrocardiographic finding in which the QT interval corrected for heart rate using Fridericia's formula. QTc = QT/∛(RR/1000)

RR = Respiration rate

QT Interval: Ventricular depolarization plus ventricular repolarization Normal Range: 400 to 460 msec
From first dose to end of study, (approximately 260 weeks)
Post-exercise Left Ventricular Outflow Tract (LVOT) Gradient Over Time
Time Frame: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
Number of participants with changes of Post-exercise left ventricular outflow tract (LVOT) gradient over time
At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
Resting Left Ventricular Outflow Tract (LVOT) Gradient Over Time
Time Frame: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
Resting left ventricular outflow tract (LVOT) gradient over time
At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
Post Valsalva Left Ventricular Outflow Tract (LVOT) Gradient Over Time
Time Frame: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
Post Valsalva left ventricular outflow tract (LVOT) gradient over time
At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252
Participants With >= 1 NYHA Function Class Improvement
Time Frame: 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

Participants with >= 1 NYHA function class improvement.

The NYHA Functional Classification of heart failure assigns participants to 1 of 4 categories based on the participant's symptoms.

Class 1: No limitation of physical activity. Ordinary physical activity does not cause undue fatigue, palpitation, dyspnea (shortness of breath).

Class 2: Slight limitation of physical activity. Comfortable at rest. Ordinary physical activity results in fatigue, palpitation, dyspnea (shortness of breath).

Class 3: Marked limitation of physical activity. Comfortable at rest. Less than ordinary activity causes fatigue, palpitation, or dyspnea.

Class 4: Unable to carry on any physical activity without discomfort. Symptoms of heart failure at rest. If any physical activity is undertaken, discomfort increases.
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
Mean Change From Baseline in the Overall KCCQ PRO Score.
Time Frame: 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
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a self-administered 23-item questionnaire questionnaire that measure the participant's perception of their health status, including their heart failure (HF) symptoms, impact on physical and social function and how their HF impacts the quality of life. KCCQ quantifies 7 domains: physical limitations (6 items), symptom stability (1 item), symptom frequency (4 items), symptom burden (3 items), self-efficacy (2 items), quality of life (3 items) and social limitations (4 items). Scores were generated for each domain and scaled from 0 to 100, with 0 denoting the worst and 100 the best possible status. Overall KCCQ Pro score is the average of all the domains, symptom frequency and symptom burden scores, and transformed to a single score which ranged from 0 (worst) to 100 (the best possible status), where the higher score reflected better health status.
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
Mean Change From Baseline in Serum NT-proBNP.
Time Frame: 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
Mean change from baseline in Serum N-terminal pro B-type natriuretic peptide levels.
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
Number of Participants Who Received Septal Reduction Therapy
Time Frame: 252 weeks
Number of participants who received septal reduction therapy
252 weeks
Plasma Concentration of Mavacamen Overtime
Time Frame: 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
Plasma concentration of Mavacamen overtime
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Bristol-Myers Squibb

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 26, 2018

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 9, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 9, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 5, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 5, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 12, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 6, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
January 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CV027-008

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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