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A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy

Mise à jour : Il y a 4 ans
Référence : EUCTR2014-002072-92

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Extrait

- To determine the safety and tolerability of multiple ascending repeat IV doses of PF-06252616 in ambulatory boys with DMD. - To demonstrate the efficacy of treatment with IV doses of PF-06252616 based on an observed mean change from baseline on function (4 Stair Climb) as compared to placebo following 49 weeks of treatment.

Critère d'inclusion

  • Duchenne's Muscular Dystrophy

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