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Queen Square Centre for Neuromuscular Diseases

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A Phase Ib/II, trial of PF-06252616 in ambulatory boys with Duchenne Muscular Dystrophy.

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).