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A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Ambulatory Boys with Duchenne Muscular Dystrophy

Principal Investigator:
Kathryn
Wagner

This is a Phase 2 randomized, two-period, double-blind, placebo-controlled, multiple ascending dose study to evaluate the safety, efficacy, pharmacokinetic, and pharmacodynamic of PF-06252616 administered to ambulatory boys diagnosed with DMD ages 6 to <10 years of age. Eligible participants will be randomly assigned to 1 of 3 groups for approximately 96 weeks (two treatment periods of 48 weeks). Three IV infused dose levels (5, 20, 40 mg/kg) administered every 28 days will be investigated in a within subject dose escalating fashion. At each dose level, dosing will be administered via a 2-hour IV infusion every 4 weeks for a total of 16 weeks (4 doses).

The study drug, PF-06252616, will be evaluated in its ability to reduce the levels of muscle growth inhibitor, myostatin, to increase muscle mass and function in DMD males.

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