Drisapersen extension study
Research type
Research Study
Full title
An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy.
IRAS ID
189216
Contact name
Francesco Muntoni
Contact email
Sponsor organisation
BioMarin Pharmaceutical Inc.
Eudract number
2015-001955-54
Duration of Study in the UK
1 years, 11 months, 30 days
Research summary
Duchennes Muscular Dystrophy (DMD) is a genetically inherited disease linked to the X chromosome and affects approximately 1 in 3500 newborn boys. DMD is Life Limiting and due to it's symptoms being delayed motor milestones and being able to run and jump properly, most are diagnosed at about age 5. If left untreated, most boys will require the use of a wheelchair before their teens.
The purpose of this study is to provide re-access to drisapersen for patients who previoulsy participated in drisapersen studies and cannot take part in ongoing studies. Participants will be provided drisapersen until it is commercially available in their country.
Participants will receive drisapersen administered either as continuous 6mg/kg/week via subcutaneous injection, intermittent 6mg/kg/week for 8 weeks followed by 4 weeks no dosing or intravenous infusion of a maximum 6mg/kg/week over a 2 hour infusion.
The duration of participation will cary dependant upon, regulatory approval/refusal, patients having serious adverse events and having to withdraw from the study or if the sponsor withdraws due to safety and efficacy findings.Study sites: this study is to be conducted at approximately 90 sites worldwide.
REC name
London - Riverside Research Ethics Committee
REC reference
15/LO/1657
Date of REC Opinion
26 Nov 2015
REC opinion
Further Information Favourable Opinion