SIDEROS-E
Research type
Research Study
Full title
A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study
IRAS ID
252024
Contact name
Michela Guglieri
Contact email
Sponsor organisation
Santhera Pharmaceuticals (Switzerland) Limited
Eudract number
2017-004279-30
Duration of Study in the UK
years, 19 months, 0 days
Research summary
The purpose of this study is to evaluate the long-term safety and efficacy of idebenone in Duchenne Muscular Dystrophy (DMD) patients who have previously completed the SIDEROS study. DMD is the most common and devastating type of muscular wasting condition with no available treatment option. It affects 1 in 3500–5000 males born worldwide, DMD is an orphan disease. The progression of the disease leads to difficulties in functioning and death, with patients frequently leading to respiratory and cardiac failure in their late twenties. Typically, the progressive loss of respiratory function end up into respiratory insufficiency and life-threatening lung infections during the second or third decade of life. Santhera are conducting a large study known as SIDEROS to assess the efficacy of idebenone in delaying the loss of respiratory function, whilst also monitoring safety and tolerability of idebenone in at least 266 DMD patients. SIDEROS study treatment period is 18 months. The patients who have successfully completed SIDEROS study will have the opportunity to enter this proposed study extension, SIDEROS-E. The primary objective of the extension study is to assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. The secondary objective is to describe the long-term respiratory function in patients who used idebenone and completed the SIDEROS study.
Participants will undergo 5 study visits and will receive medication to swallow 3 times daily at home. 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 - Tyne & Wear South Research Ethics Committee
REC reference
18/NE/0292
Date of REC Opinion
26 Oct 2018
REC opinion
Further Information Favourable Opinion