Non-obstructive Hypertrophic CardiomyopathyPhase 2MAVERICK-HCMNCT03442764
A Phase 2 Study of Mavacamten in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)
A Phase 2 study testing mavacamten in people with Non-obstructive Hypertrophic Cardiomyopathy, run by MyoKardia, Inc. at 32 sites in United States.
Trial summary
This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten doses titrated to achieve 1 of 2 target drug concentrations. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Non-obstructive Hypertrophic Cardiomyopathy
- What is being tested?
- mavacamten
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 59 participants
- Where
- 32 sitesUnited States
- Study dates
- 30 Mar 2018to 7 Jan 2020
- 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
- Diagnosed with nHCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15mm at Screening or ≥ 13mm with a positive family history of HCM.
- Age 18 and greater, Body weight > 45kg
- Documented LVEF ≥ 55% at the Screening as determined by echo central lab
- LVOT gradient < 30 mmHg at rest AND during Valsalva AND post-exercise
- NYHA functional class II or III
- Elevated NT-proBNP at rest
Who cannot join
- History of syncope, sustained ventricular tachyarrhythmia with exercise, obstructive coronary artery disease or myocardial infarction within the past 6 months
- History of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator (ICD) discharge within 6 months prior to Screening
- Current treatment with disopyramide or ranolazine (within 14 days prior to Screening)
- Current or planned treatment during the study with a combination of beta-blockers and calcium channel blockers
- Has been treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening
- History of resting or post-exercise LVOT >30 mmHg unless subsequently treated by septal reduction
Show all 10Show fewer
- Has QTc Fridericia (QTcF) >480 ms or any other ECG abnormality considered by the investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II)
- Has persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate-controlled within 1 year of Screening
- History of clinically significant malignant disease within 10 years such as non-metastatic cutaneous squamous cell or basal cell carcinoma
- History or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator or MyoKardia physician, would pose a risk to subject safety or interfere with the study evaluation, procedures, or completion.
Trial locations and contacts
32 sites in 1 country.
32 sites in 1 country
United States32 sites
Michigan Medicine
Ann Arbor, Michigan, 48109
University of Maryland Medical System
Baltimore, Maryland, 21201
St. Luke's Cardiology Associates
Bethlehem, Pennsylvania, 18018
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Carolinas Medical Center
Charlotte, North Carolina, 28203
University of Virginia
Charlottesville, Virginia, 22908
University of Cincinnati Medical Center
Cincinnati, Ohio, 45219
The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390-9034
Henry Ford Hospital
Detroit, Michigan, 48202
Duke Cardiology at Southpoint
Durham, North Carolina, 27713
Northwestern University
Evanston, Illinois, 60208
Penn State Health Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033-0850
Baylor St. Luke Medical Center at Houston, Texas Heart Institute Out-patient Clinic
Houston, Texas, 77030
Houston Methodist Hospital
Houston, Texas, 77030
St. Vincent Medical Group
Indianapolis, Indiana, 46260
University of Iowa Hospitals and clinics
Iowa City, Iowa, 52242
Cedars-Sinai Medical Center (Smidt Heart Institute)
Los Angeles, California, 90048
Unity Point Health Meriter Heart and Vascular Institute
Madison, Wisconsin, 53713
NYU Winthrop Hospital
Mineola, New York, 11501
Intermountain Medical Center
Murray, Utah, 84107
Yale New Haven Hospital
New Haven, Connecticut, 06520
NYU Langone Medical Center
New York, New York, 10016
Stanford Hospital and Clinics/Stanford University
Palo Alto, California, 94305
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
UPMC Presbyterian
Pittsburgh, Pennsylvania, 15213
Oregon Health and Science University
Portland, Oregon, 97239
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Utah Medical Center
Salt Lake City, Utah, 84132
University of California, San Francisco
San Francisco, California, 94143
Mayo Clinic Arizona
Scottsdale, Arizona, 85259
University of Washington Medical Center
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Concentration-guided, Exploratory Study of Mavacameten in Patients With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) and Preserved Left Ventricular Ejection Fraction
This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten doses titrated to achieve 1 of 2 target drug concentrations.
- Being tested
- mavacamten, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mavacamtenDrug
Also called MYK-461
MYK-461
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Group 1 | Experimental | Active Treatment for participants with base target trough concentration Receives: Drug: mavacamten |
| Group 2 | Experimental | Active Treatment for participants with higher target trough concentration Receives: Drug: mavacamten |
| Placebo | Placebo comparator | Placebo Group Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)
This is the percentage of participants who experienced at least one treatment emergent adverse event (TEAE)
Measured: From first dose to 8 weeks following last dose (Up to 24 weeks)
Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE)
This is the percentage of participants who experienced at least one serious treatment-emergent adverse event (STEAE)
Measured: From first dose to 8 weeks following last dose (Up to 24 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Feb 2018
- Study start30 Mar 2018
- Primary completion7 Jan 2020
- Study completion7 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Non-obstructive Hypertrophic CardiomyopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 59 participants (actual)
Sponsor and investigators
- Lead sponsor
- MyoKardia, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Myokardia Medical Information Team, Study director, MyoKardia, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03442764
- Sponsor's protocol ID
- MYK-461-006
- Acronym
- MAVERICK-HCM
- Status last verified
- Jul 2022
- First submitted
- 9 Feb 2018
- Last updated
- 9 Aug 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ho CY, Mealiffe ME, Bach RG, Bhattacharya M, Choudhury L, Edelberg JM, Hegde SM, Jacoby D, Lakdawala NK, Lester SJ, Ma Y, Marian AJ, Nagueh SF, Owens A, Rader F, Saberi S, Sehnert AJ, Sherrid MV, Solomon SD, Wang A, Wever-Pinzon O, Wong TC, Heitner SB. Evaluation of Mavacamten in Symptomatic Patients With Nonobstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2020 Jun 2;75(21):2649-2660. doi: 10.1016/j.jacc.2020.03.064.
PubMed 32466879
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Concentration-guided, Exploratory Study of Mavacameten in Patients With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM) and Preserved Left Ventricular Ejection Fraction
- ClinicalTrials.gov ID
- NCT03442764
- Acronym / sponsor ID
- MAVERICK-HCM / MYK-461-006
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 59 participants (actual)
- Lead sponsor
- MyoKardia, Inc.Industry (pharma, biotech, devices)
- Study start
- 30 Mar 2018 (actual)
- Primary completion
- 7 Jan 2020 (actual)
- Study completion
- 7 Jan 2020 (actual)
- First posted
- 22 Feb 2018 (actual)
- Results
- Posted 9 Aug 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a multicenter, exploratory, randomized, double-blind study of the administration of mavacamten in 60 participants with symptomatic nHCM randomized to receive a 16-week course of mavacamten doses titrated to achieve 1 of 2 target drug concentrations.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1 | Experimental | Drug: mavacamtenActive Treatment for participants with base target trough concentration |
| Group 2 | Experimental | Drug: mavacamtenActive Treatment for participants with higher target trough concentration |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo Group |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Diagnosed with nHCM (hypertrophied and non-dilated left ventricle in absence of systemic or other known cause), with LV wall thickness ≥ 15mm at Screening or ≥ 13mm with a positive family history of HCM.
- Age 18 and greater, Body weight > 45kg
- Documented LVEF ≥ 55% at the Screening as determined by echo central lab
- LVOT gradient < 30 mmHg at rest AND during Valsalva AND post-exercise
- NYHA functional class II or III
Key exclusion criteria (10 listed)
- History of syncope, sustained ventricular tachyarrhythmia with exercise, obstructive coronary artery disease or myocardial infarction within the past 6 months
- History of resuscitated sudden cardiac arrest at any time or known appropriate implantable cardioverter defibrillator (ICD) discharge within 6 months prior to Screening
- Current treatment with disopyramide or ranolazine (within 14 days prior to Screening)
- Current or planned treatment during the study with a combination of beta-blockers and calcium channel blockers
- Has been treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| mavacamtenAlso: MYK-461MYK-461 | Drug | Group 1, Group 2 |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE)
Time frame: From first dose to 8 weeks following last dose (Up to 24 weeks)
This is the percentage of participants who experienced at least one treatment emergent adverse event (TEAE)
Percentage of Participants Who Experienced at Least One Serious Treatment-emergent Adverse Event (STEAE)
Time frame: From first dose to 8 weeks following last dose (Up to 24 weeks)
This is the percentage of participants who experienced at least one serious treatment-emergent adverse event (STEAE)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 32466879Ho CY, Mealiffe ME, Bach RG, Bhattacharya M, Choudhury L, Edelberg JM, Hegde SM, Jacoby D, Lakdawala NK, Lester SJ, Ma Y, Marian AJ, Nagueh SF, Owens A, Rader F, Saberi S, Sehnert AJ, Sherrid MV, Solomon SD, Wang A, Wever-Pinzon O, Wong TC, Heitner SB. Evaluation of Mavacamten in Symptomatic Patients With Nonobstructive Hypertrophic Cardiomyopathy. J Am Coll Cardiol. 2020 Jun 2;75(21):2649-2660. doi: 10.1016/j.jacc.2020.03.064.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Myokardia Medical Information Team study director
MyoKardia, Inc.
32 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing mavacamten in people with Non-obstructive Hypertrophic Cardiomyopathy, run by MyoKardia, Inc. at 32 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 6 inclusion and 10 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 Aug 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 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. Some participants in this study may receive a placebo. 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 Aug 2022). 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?
Percentage of Participants Who Experienced at Least One Treatment Emergent Adverse Event (TEAE) (time frame: From first dose to 8 weeks following last dose (Up to 24 weeks)). The study lists 2 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 NCT03442764, 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 Aug 2022.