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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:

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.

Bradley L. Schlaggar, M.D., Ph.D., Named President and CEO of Kennedy Krieger Institute

We’re thrilled to welcome Bradley L. Schlaggar, M.D., Ph.D., to the Kennedy Krieger family as our next President and CEO.
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