Queen Square Centre for Neuromuscular Diseases



A phase IIb, study of PRO045 in subjects with Duchenne muscular dystrophy (DMD)

Sponsor Prosensa
Funder Prosensa
CI Professor Volker Straub
sites Newcastle and London GOSH

Newcastle: becky.davis@ncl.ac.uk

London: c.uzowuru@ucl.ac.uk

More information

Primary objective

To assess the efficacy of the drug (PRO045) after 48 weeks treatment in ambulant patient with DMD.

Secondary objectives

  • 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.