SIDEROS, V1
Research type
Research Study
Full title
A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids
IRAS ID
214375
Contact name
Michela Guglieri
Contact email
Sponsor organisation
Santhera Pharmaceuticals (Switzerland) Limited
Eudract number
2016-000602-10
Duration of Study in the UK
2 years, 10 months, 13 days
Research summary
Duchenne Muscular Dystrophy (DMD) is a condition that affects about 1 in every 3000-5000 boys. The disease affects the muscles, including muscles involved in breathing and the heart. This clinical research study is to be conducted in the patients with DMD to find out more information about the efficacy of idebenone in slows down the decline of respiratory function.
Nerve and muscle cells, including lung muscle cells, are particularly energy-demanding and are, therefore, more prone to rapid loss of function or death when the mitochondria ("cellular power plants") do not produce enough energy. By preserving the function of the mitochondria and protecting cells from oxidative damage, it is believed that idebenone can prevent cell damage and increase the production of energy. Idebenone is a small molecule which facilitates the transport of electrons within mitochondria, and thus helps with the production of cellular energy. In this study, idebenone will be tested in order to determine its effect on breathing function in DMD patients receiving Glucocorticoid Steroids compared to placebo and to see how well idebenone is tolerated over an 18months treatment period. This will be assessed by a series of physical tests and laboratory assessments which will also evaluate any side effects. These will be described in more detail in session A13.
This study will be conducted at sites that are specialised in treating subjects with Duchenne muscular dystrophy. Participants will undergo 10 study visits (Breathing test, physical examinations, heart test and samples being taken). They will receive oral medication to swallow 3 times a day at home alongside with a participant dairy and handheld breathing tester for a short 5-minute test to be performed at home once a week.
REC name
North East - York Research Ethics Committee
REC reference
16/NE/0370
Date of REC Opinion
28 Dec 2016
REC opinion
Further Information Favourable Opinion