A Long-term Safety Extension Study of Mavacamten (MYK-461) in Adults with Hypertrophic Cardiomyopathy Who Have Completed the MAVERICKHCM (MYK-461-006) or EXPLORER-HCM (MYK-461-005) Trials (MAVA-LTE)
- Conditions
- Hypertrophic cardiomyopathyMedDRA version: 20.0Level: PTClassification code: 10020871Term: Hypertrophic cardiomyopathy Class: 100000004850Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
- Registration Number
- CTIS2022-502858-14-00
- Lead Sponsor
- Myokardia Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 250
Has completed the Parent Study through to the EOS Visit within 90 days of signing consent. (Participants who are beyond the 90-day window from the EOS Visit may be included in this study pending MyoKardia Medical Monitor approval). Participants who prematurely discontinued from the Parent Study or the MAVA-LTE Study may be considered for inclusion., Is able to understand and comply with the study procedures, understand the risks involved in the study, and provide informed consent according to federal, local, and institutional guidelines before the first study-specific procedure, Body weight is greater than 45 kg at the Screening Visit or Day 1 (Day 1 weight must be verified prior to dosing), Has adequate acoustic windows to enable accurate TTEs (refer to the Echocardiography Site Instruction Manual), Has documented LVEF = 50% by echocardiography core laboratory read of screening TTE at rest, Has safety laboratory parameters within normal limits (according to the central laboratory reference range); however, a participant with safety laboratory parameters outside normal limits may be included if he or she meets all of the following criteria: • The safety laboratory parameter outside normal limits is considered by the Investigator to be clinically unimportant • If there is an alanine aminotransferase or aspartate aminotransferase result, the value must be < 3 × the upper limit of the laboratory reference range • The body size–adjusted estimated glomerular filtration rate is = 30 mL/min/1.73 m2, Female participants must not be pregnant or lactating and, if sexually active, must use one of the following highly effective birth control methods from the Screening Visit through 4 months after the last dose of investigational medicinal product (IMP) • combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation or progestogen-only hormonal contraception associated with inhibition of ovulation by oral, implantable, or injectable route of administration • intrauterine device (IUD) •intrauterine hormone-releasing system (IUS) • bilateral tubal occlusion • Female is surgically sterile for 6 months or postmenopausal for 1 year. Permanent sterilization includes hysterectomy, bilateral oophorectomy, bilateral salpingectomy, and/or documented bilateral tubal occlusion at least 6 months prior to Screening. Females are considered postmenopausal if they have had amenorrhea for at least 1 year or more following cessation of all exogenous hormonal treatments, and follicle-stimulating hormone levels are in the postmenopausal range. In addition to the above contraceptive requirements for female participants, male partners must also use a contraceptive (eg, barrier, condom, or vasectomy), Sub-Study Inclusion Criteria: Each participant must meet the inclusion/exclusion criteria and be enrolled in the MYK-461-007 Study. Participants from the MAVERICK-HCM Study may participate in the CMR substudy. Participants from the EXPLORER-HCM Study must have already participated in the CMR substudy.
Has persistent or permanent atrial fibrillation, not on anticoagulation for at least 4 weeks prior, and/or is not adequately rate-controlled (Note: participants with persistent or permanent atrial fibrillation who are anticoagulated and adequately rate-controlled are allowed), Has any ECG abnormality considered by the Investigator to pose a risk to participant safety (eg, second-degree atrioventricular block type II), Has documented obstructive coronary artery disease (> 70% stenosis in one or more epicardial coronary arteries) or history of myocardial infarction, Has known moderate or severe (as per Investigator’s judgment) aortic valve stenosis at Screening Visit, Has hypersensitivity to any of the components of the mavacamten formulation, Has participated in a clinical trial in which the participant received any investigational drug (or is currently using an investigational device) within 30 days prior to Screening, or at least 5 times the respective elimination half-life (whichever is longer), except for participation in MAVERICK-HCM or EXPLORER-HCM. Prior participation in a non-interventional observational study is allowed., Has a history of syncope or a history of sustained ventricular tachyarrhythmia with exercise between Parent Study EOS Visit and Screening Visit., Has a history of resuscitated sudden cardiac arrest or known history of appropriate implantable cardioverter-defibrillator (ICD) discharge for lifethreatening ventricular arrhythmia between Parent Study EOS Visit and Screening Visit. (Note: history of anti-tachycardia pacing is allowed), Sub-Study Exclusion Criteria: An ICD or pacemaker. Starting with Protocol Amendment 4, participants must not have a device that is incompatible with MRI. ? Atrial fibrillation at the time of scheduled day of CMR (Note: See Appendix 5 for Germany-specific requirements, starting with Amendment 3.2 G)., Currently treated with disopyramide or ranolazine (within 14 days prior to Screening Visit) or treatment with disopyramide or ranolazine is planned during the study, Currently treated or planned treatment during the study with a combination of beta blocker and verapamil or a combination of beta blocker and diltiazem, Has any acute or serious comorbid condition (eg, major infection or hematologic, renal, metabolic, gastrointestinal, or endocrine dysfunction) that, in the judgment of the Investigator, could lead to premature termination of study participation or interfere with the measurement or interpretation of the efficacy and safety assessments in the study, History of clinically significant malignant disease that developed since enrollment in the Parent Study ? Participants who have been successfully treated for nonmetastatic cutaneous squamous cell or basal cell carcinoma or have been adequately treated for cervical carcinoma in situ or breast ductal carcinoma in situ can be included in the study, Is unable to comply with the study requirements, including the number of required visits to the clinical site, s employed by or is a relative of someone employed by MyoKardia, the Investigator, or his/her staff or family, An ICD or pacemaker. Starting with Protocol Amendment 4, participants must not have a device that is incompatible with MRI. • Atrial fibrillation at the time of scheduled day of CMR (Note: See Appendix 5 for Germany-specific requirements, start
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method