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| Sponsor | PFIZER |
| Funder | PFIZER |
| CI | Professor Kate Bushby |
| sites | Newcastle and London GOSH |
| Contact |
Newcastle: becky.davis@ncl.ac.uk ; London: c.uzowuru@ucl.ac.uk |
| More information on the trial |
Study information
This is a Phase II randomised, 2-period, double-blind, placebo-controlled, multiple ascending dose study.
Primary objective
To assess the safety, efficacy, pharmacokinetics and pharmacodynamics of the drug (PF-06252616) given to ambulatory boys diagnosed with DMD.
Three IV infused doses (5, 20, 40 mg/kg) given every 28 days will be investigated in a within patient dose increasing fashion.
Recruitment
Approximately 105 eligible patients will be randomly assigned to one of three sequence groups.
It will last about 96 weeks (two treatment periods of approximately 48 weeks each).