Study type

Study topic

Human medicinal product

Study type

Non-interventional study

Data collection methods

Secondary use of data
Non-interventional study

Non-interventional study design

Other
Systematic review and meta-analysis
Study drug and medical condition

Name of medicine

CAMZYOS

Name of medicine, other

mavacamten

Anatomical Therapeutic Chemical (ATC) code

(C01EB) Other cardiac preparations
Other cardiac preparations

Medical condition to be studied

Hypertrophic cardiomyopathy
Population studied

Short description of the study population

The source population is defined by the parent study protocols, ie, adults 18 years of age or older with symptomatic HCM (oHCM and/or nHCM) in existing Phase 3 and 3b/4 studies.

Age groups

Adult and elderly population (≥18 years)
Adults (18 to < 65 years)
Adults (18 to < 46 years)
Adults (46 to < 65 years)
Elderly (≥ 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

964
Study design details

Main study objective

To assess the risk of major CV events observed under mavacamten treatment is non-inferior to the risk presented under placebo treatment.

Outcomes

- Time from first dose to the first occurrence of an e-MACE event. e-MACE event defined as a composite of adjudicated events of CV death, non-fatal myocardial infarction (MI), non-fatal stroke, hospitalization for heart failure (HF), hospitalization for arrhythmia, other CV hospitalization (for events other than heart failure or arrhythmia), or appropriate shock therapy from implanted cardiac device (ICD).
- The first occurrence of MACE (4-point), where MACE is defined as a composite of adjudicated event of CV death, non-fatal MI, non-fatal stroke, or hospitalization for HF.
- The first occurrence of MACE-plus, where MACE-plus is defined as a composite of adjudicated event of CV death, non-fatal MI, non-fatal stroke, hospitalization for HF, or hospitalization for arrhythmia, or appropriate shock therapy from ICD.
- Time from first dose to all-cause mortality.
- Time from first dose to CV death.
- Time from first dose to the first occurrence of non-fatal MI.
- Time from first dose to the first occurrence of non-fatal stroke.
- Time from first dose to the first occurrence of hospitalization for HF.
- Time from first dose to the first occurrence of hospitalization for arrhythmia.
- Time from first dose to the first occurrence of other CV hospitalization (for events other than HF or arrhythmia).
- Time from first dose to the first occurrence of appropriate shock therapy from ICD.

Data analysis plan

This meta-analysis will combine evidence using patient-level data from these studies (EXPLORER-HCM, VALOR-HCM, EXPLORER-CN, MEMENTO trials and ODYSSEY-HCM) and appropriate statistical methods will be applied, to allow inference to be made to the population of symptomatic HCM patients. This meta-analysis will be performed within 1 year after the last study is unblinded.