RIM4DMD
Research type
Research Study
Full title
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
IRAS ID
185578
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
espeRare Foundation
Eudract number
2015-002530-50
Duration of Study in the UK
1 years, 3 months, 0 days
Research summary
This study examines the safety and tolerability and effects on the muscles of a new drug, rimeporide, in boys aged 6 to 14 years with Duchenne Muscular Dystrophy (DMD), a rare hereditary disorder for which new treatments are urgently needed.
In 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. Rimeporide has a positive effect on the levels of sodium and calcium in the muscle cells, by blocking an important protein called NHE-1, hoped to slowing of the progressive loss of strength of the muscles. Results of studies in animals have been encouraging and the drug has been granted designation as an Orphan Drug in Europe.
Rimeporide has been given already to 145 healthy adult volunteers and 21 adult patients with heart failure and so far appears well tolerated. However, before large-scale tests of rimeporide in DMD can be conducted it is necessary to examine the safety and tolerability in children with DMD
The study will enrol 20 patients with DMD, aged 6 to 14 years. 4 dose levels will be tested, with 5 patients taking the drug at each dose level. Treatment at the each level can only commence if safety at the lower level is judged satisfactory.
Each patient will receive rimeporide for 4 weeks and the patients will be followed up for an additional two weeks after the last dose of rimeporide. Treatment will be administered orally three times daily. Patients will be carefully followed up for safety and will attend the study centre on 4 occasions. Effects on biomarkers of DMD will be explored, including optional assessment by nuclear magnetic resonance techniques conducted at the Institute of Myology in Paris.REC name
London - Riverside Research Ethics Committee
REC reference
15/LO/1432
Date of REC Opinion
23 Oct 2015
REC opinion
Further Information Favourable Opinion