Rimeporide in Patients With Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2018-02-01
Target enrollment:
Participant gender:
Summary
In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and
sodium in the muscles cells which is thought to be important in the damage which occurs
overtime. Sodium/proton type 1 exchanger (NHE-1) inhibition is an innovative pathway that has
proved to efficiently prevent the accumulation of muscle damage (inflammation and fibrosis)
in animal models of muscular dystrophies and heart failure. Based on prior safety and
efficacy results in animal and humans, NHE-1 inhibition with Rimeporide represents a new
therapeutic approach with no restriction on age and on genetic subtypes which could be
combined to other treatments that restore or augment dystrophin.This study examines the
safety and tolerability and effects on the muscles of rimeporide, in patients aged 6 to 14
years with Duchenne Muscular Dystrophy (DMD).