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Hypertrophic CardiomyopathyPhase 2PIONEER-OLENCT03496168

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

A Phase 2 study testing mavacamten in people with Hypertrophic Cardiomyopathy, run by Bristol-Myers Squibb at 4 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Hypertrophic Cardiomyopathy
What is being tested?
mavacamten
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
13 participants
Where
4 sitesUnited States
Study dates
26 Apr 2018to 9 Nov 2023
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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

Trial locations and contacts

4 sites in 1 country.

United States4 sites

  • Local Institution - 0004

    Durham, North Carolina, 27710

  • Local Institution - 0001

    New Haven, Connecticut, 06520-8017

  • Local Institution - 0002

    Portland, Oregon, 97239

  • Local Institution - 0003

    Scottsdale, Arizona, 85259

Other trials in United States

About this study

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)

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).

Being tested
mavacamten
Keywords
Symptomatic, Obstructive, Left ventricular outflow tract gradient
Medical subject headings
Cardiomyopathy, Hypertrophic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • mavacamtenDrug

    Also called MYK-461

    mavacamten capsules

Study groups in NCT03496168
Study groupRoleWhat they receive
mavacamten (MYK-461)ExperimentalNot described
Receives: Drug: mavacamten

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Treatment Emergent Adverse Events and Treatment Emergent Serious Adverse Events

    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.

    Measured: From first dose to end of treatment + 56 days (Approximately an average of 240 Weeks)

  2. Number of Participants Who Had Cardiovascular Death

    Number of participants who had died due to cardiovascular reasons.

    Measured: From first dose to end of study, (approximately 260 weeks)

  3. Number of Participants Who Experienced Sudden Death

    Number of participants who experienced sudden death

    Measured: From first dose to end of study, (approximately 260 weeks)

  4. Number of Participants Who Were Hospitalized for Cardiovascular Reasons.

    Number of participants who were hospitalized for cardiovascular reasons.

    Measured: From first dose to end of study, (approximately 260 weeks)

  5. Number of Participants With Heart Failure Due to Systolic Dysfunction, Defined as Asymptomatic LVEF < 50%

    Number of participants with heart failure due to systolic dysfunction, defined as asymptomatic LVEF < 50%

    Measured: From first dose to end of study, (approximately 260 weeks)

  6. Number of Participants With LVEF < 50% as Measured by Echocardiography.

    Number of participants with LVEF < 50% as measured by echocardiography.

    Measured: From first dose to end of study, (approximately 260 weeks)

  7. Number of Participants Who Were Experienced Myocardial Infarction

    Number of participants who experienced myocardial infarction.

    Measured: From first dose to end of study, (approximately 260 weeks)

  8. Number of Participants With Ventricular Arrhythmias.

    Types of Ventricular Arrhythmias measured in this endpoint will be:

    Ventricular Tachycardia Ventricular Fibrilation Ventricular Flutter

    Measured: From first dose to end of study, (approximately 260 weeks)

  9. Number of Participants Who Experienced Syncope

    Number of participants who experienced syncope.

    Syncope will be defined as participants who experienced dizziness or orthostatic hypotension.

    Measured: From first dose to end of study, (approximately 260 weeks)

  10. Number of Participants Who Experienced Seizures

    Number of participants who were experienced seizures.

    Measured: From first dose to end of study, (approximately 260 weeks)

  11. Number of Participants Who Were Experienced Strokes

    Number of participants who were experienced strokes.

    Measured: From first dose to end of study, (approximately 260 weeks)

  12. Number of Participants With a Change in QT and QTcF Intervals.

    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

    Measured: From first dose to end of study, (approximately 260 weeks)

  13. Post-exercise Left Ventricular Outflow Tract (LVOT) Gradient Over Time

    Number of participants with changes of Post-exercise left ventricular outflow tract (LVOT) gradient over time

    Measured: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252

  14. Resting Left Ventricular Outflow Tract (LVOT) Gradient Over Time

    Resting left ventricular outflow tract (LVOT) gradient over time

    Measured: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252

  15. Post Valsalva Left Ventricular Outflow Tract (LVOT) Gradient Over Time

    Post Valsalva left ventricular outflow tract (LVOT) gradient over time

    Measured: At Baseline, Week 4, Week 48, Week 72, Week 156, Week 204 and Week 252

  16. Participants With >= 1 NYHA Function Class Improvement

    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.

    Measured: 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

  17. Mean Change From Baseline in the Overall KCCQ PRO Score.

    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.

    Measured: 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

  18. Mean Change From Baseline in Serum NT-proBNP.

    Mean change from baseline in Serum N-terminal pro B-type natriuretic peptide levels.

    Measured: 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

  19. Number of Participants Who Received Septal Reduction Therapy

    Number of participants who received septal reduction therapy

    Measured: 252 weeks

  20. Plasma Concentration of Mavacamen Overtime

    Plasma concentration of Mavacamen overtime

    Measured: 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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Apr 2018
  2. Study start26 Apr 2018
  3. Primary completion9 Nov 2023
  4. Study completion9 Nov 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
13 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03496168
Sponsor's protocol ID
CV027-008
Acronym
PIONEER-OLE
Status last verified
Jan 2025
First submitted
5 Apr 2018
Last updated
9 Jan 2025
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Masri A, Lester SJ, Stendahl JC, Hegde SM, Sehnert AJ, Balaratnam G, Shah A, Fox S, Wang A. Long-Term Safety and Efficacy of Mavacamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy: Interim Results of the PIONEER-OLE Study. J Am Heart Assoc. 2024 Apr 16;13(8):e030607. doi: 10.1161/JAHA.123.030607. Epub 2024 Apr 9.

    PubMed 38591260

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing mavacamten in people with Hypertrophic Cardiomyopathy, run by Bristol-Myers Squibb at 4 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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)). The study lists 20 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03496168, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jan 2025.

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