Duchenne Muscular Dystrophy and Treatment With PF-06252616 – Phase II

Study to Evaluate the Safety, Efficacy, and Pharmacodynamics of PF-06252616 in Ambulatory Boys With Duchenne Muscular Dystrophy

Protocol Description

This multicenter international study will help researchers learn about the safety and effects of PF-06252616 on male children with Duchenne muscular dystrophy (DMD), a disease that damages the muscles causing muscle weakness and loss of the ability to walk. PF-06252616 is an engineered protein, called a monoclonal antibody, designed to inhibit a naturally occurring protein called myostatin that has a role in regulating muscle growth. Researchers believe that PF-06252616 may prevent myostatin from adversely limiting the size of muscles in people with DMD and may provide improved function.

Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, the study is accepting boys, ages 6 through 15, who have DMD and are ambulatory (can walk).
Boys: 6 through 15 years of age


Participants in this study will receive a comprehensive initial exam, including imaging, physical testing and biological samples, and then will be randomly assigned to one of three study groups. In this study, participants will receive intravenous dosages of the study drug, provided at escalating dose levels up to a maximum. Some participants will receive a placebo instead of the study drug during portions of the study.
Visits: About 28 (every 4 weeks)
Duration: 26 months

Status: Open to Enrollment

Source(s) of Support


Primary Investigator

Hoda Abdel-Hamid, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Jennifer Monahan, Clinical Research Coordinator