A phase IIb, study of PRO045 in subjects with Duchenne muscular dystrophy (DMD)
|CI||Professor Volker Straub|
|sites||Newcastle and London GOSH|
To assess the efficacy of the drug (PRO045) after 48 weeks treatment in ambulant patient with DMD.
- To assess the safety and tolerability in all study patients including those from the dose-escalation phase of the study.
- To determine the pharmacokinetics at different doses after subcutaneous administration in the patients.
- To determine the pharmacokinetics and pharmacodynamics at different doses
- after subcutaneous administration
- single intravenous dose administration
- To assess efficacy in all subjects with DMD not included in the primary objective after 48 weeks of treatment.