MAVA-LTE
Research type
Research Study
Full title
A Long-term Safety Extension Study of Mavacamten (MYK 461) in Adults with Hypertrophic Cardiomyopathy Who Have Completed the MAVERICK-HCM (MYK-461-006) or EXPLORER-HCM (MYK 461 005) Trials (MAVA-LTE)
IRAS ID
265045
Contact name
Perry Elliott
Contact email
Sponsor organisation
Myokardia Inc
Eudract number
2018-004039-64
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 4 months, 26 days
Research summary
Myokardia are conducting a long-term safety extension study of Mavacamten (MYK-461) in adults with hypertrophic cardiomyopathy (HCM), who have completed the MAVERICK-HCM (MYK-461-006) or EXPLORER-HCM (MYK 461 005) trials.
HCM is a condition that causes abnormal thickening of the heart muscle. It is a genetic condition and can be passed along through families. In some cases, thickening of the wall between the two lower chambers of the heart, blocks the flow of blood through the outlet to the left ventricle when the heart contracts. This is known as left ventricular outflow tract (LVOT) obstruction and can cause chest pain with exertion, breathlessness, and blackouts.
The genetic mutations that cause HCM affect the proteins that make up the molecular motor (the sarcomere) that allows heart muscle cells to contract.
Mavacamten is a drug being studied that specifically affects one of the sarcomere proteins, cardiac myosin, reducing its ability to cause contraction of the heart muscle.This effect is reversed when the study drug is stopped. This action of the drug is predicted to reduce LVOT obstruction and to potentially reduce heart thickening with chronic treatment.
Mavacamten has been tested in humans previously, is generally safe and is well tolerated.
Approximately 60 participants are expected to enroll in this study from the MAVERICK-HCM study, and approximately 220 participants from EXPLORER-HCM study.
All participants will receive active study drug for a duration of up to 2 years, but their status (active or placebo) in the Parent Study will remain blinded (dose strength not known) as the studies will be enrolling in parallel. All participants will undergo the same assessments and visit schedule to preserve the blind of assignments in the Parent Study. Some of these procedures include; informed consent, medical history, AEs, con-meds, physical examination, vital signs, body weight, blood tests, TTE and ECG.REC name
London - London Bridge Research Ethics Committee
REC reference
19/LO/1337
Date of REC Opinion
14 Nov 2019
REC opinion
Further Information Favourable Opinion