PHOspholamban RElated CArdiomyopathy STudy - Intervention
Status:
Completed
Trial end date:
2021-10-01
Target enrollment:
Participant gender:
Summary
Phospholamban (PLN) R14del mutation carriers may develop dilated cardiomyopathy (DCM) and/or
arrhythmmogenic cardiomyopathy (ACM). Analogous to other inherited cardiomyopathies, the
natural course of the disease is age-related ("age-related penetrance"); after a
presymptomatic phase of variable length many PLN R14del-carriers progress to overt disease,
and are diagnosed with either DCM or ARVC. PLN is a regulator of the sarcoplasmic reticulum
Ca2+-ATPase (SERCA2a) pump in cardiac muscle and thereby important for maintaining Ca2+
homeostasis. Cardiac fibrosis appears to be an early manifestation of disease. The
investigators hypothesize that treatment of presymptomatic PLN R14del-carriers with
eplerenone, which by virtue of its mineralocorticoid(aldosterone)-blocking properties is a
strong antifibrotic agent, reduces disease progression and postpones onset of overt disease.
Phase:
Phase 3
Details
Lead Sponsor:
M.p. van den Berg, MD, PhD, professor in Cardiology
Collaborators:
Netherlands: CVON, CardioVascular Research Netherlands Netherlands: TCC, Trial Coordination Center UMCG The Interuniversity Cardiology Institute of the Netherlands University Medical Center Groningen ZonMw: The Netherlands Organisation for Health Research and Development