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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 |
| Contact |
Newcastle: becky.davis@ncl.ac.uk London: c.uzowuru@ucl.ac.uk |
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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.