A Phase Ib, Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Oral Doses of Rimeporide in Patients With Duchenne Muscular Dystrophy
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).
• Duchenne muscular dystrophy genetically confirmed;
• Males between 6 and 14 years old;
• Able to walk independently at least 75 meters;
• Patients on a stable dose of corticosteroids at least 6 months prior to baseline;
• Patients able to swallow capsules size 4 according to the parents and investigator opinion;
• Willing and able to comply with all protocol requirements and procedures;
• Signed informed consents by the parent(s)/legal guardian(s);
• France only: Affiliated to or a beneficiary of a social security system