VISION-DMD
A Phase IIb Randomized, Double-blind, Parallel Placebo- and Active-controlled Study to Assess the Efficacy and Safety of VBP15 in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD)VISION-DMD (Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy, DMD) is a Phase 2a and 2b randomised, double-blind, parallel placebo- and active-controlled study. It will assess the safety and toxicity (phase 2a) and safety and efficacy (phase 2b) of the orphan drug VBP15 in ambulatory boys with Duchenne muscular dystrophy (DMD), a rare recessive X-linked form of muscular dystrophy. Funded by Horizon 2020 and the sponsor, Reveragen Biopharma Limited, the project aims to advance clinical development of VBP15 as a new therapy to revolutionise care for all patients with DMD by 2020. The study will be carried out over 18 to 24 months in approximately 15 countries and 30 international study sites. The phase 2a study will be conducted at approximately 10 U.S. study sites. The phase 2b study, supported by ECRIN, will be conducted at approximately 30 international study sites in 14 different countries (UK, France, Italy, Spain, Belgium, Netherlands, Denmark, Sweden, Switzerland, Poland, Czech Republic, Turkey, Israel, and Australia).
The trial is ongoing but ECRIN's involvement ended in 2019.
Population: paediatric
