Pfizer is currently recruiting participants to enroll a phase 2 clinical trial that intends to assess the safety, efficacy and pharmacological parameters of its product, PF-06252616, in boys with Duchenne muscular dystrophy (DMD) who are able to walk (ambulatory). The clinical trial is registered under the identifier NCT02310763 and is entitled “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”.
DMD is an inherited disorder caused by a defective gene called dystrophin. It is characterized by a rapid progressive skeletal muscle weakness caused by chronic inflammation and the degeneration of muscle cells and tissue, which can compromise locomotion and respiratory function, leading to breathing complications and cardio-respiratory failure. DMD has a rapid progression and affects mainly boys. Most DMD patients require a wheelchair by the age of 12 and patients often succumb to the disease in their 20s.
PF-06252616 is an experimental monoclonal antibody against myostatin, a protein that is involved in muscle growth. In the trial, three different intravenous doses of PF-06252616 will be tested – 5, 20 and 40 mg/kg.
The primary goal of the stuy is to assess the safety and efficacy of PF-06252616 therapy in a total period of approximately 96 weeks (2 periods of 48 weeks each). Participants will be randomly divided into three groups: in the first period of 48 weeks two groups will receive intravenous infused doses of PF-06252616 monthly and the third group will receive a placebo. In the second period, the placebo group will receive PF-06252616 and the other two either the placebo or PF-06252616. Treatment efficacy will be assessed through improvements in overall motor function, pulmonary function, exercise capacity and muscle strength.
Pfizer plans to enroll up to 150 male, ambulatory individuals, aged 6 to 9 years old, diagnosed with DMD.
If you are interested in more information, please contact Pfizer at 1-800-718-1021. To access a list of the 30 study locations involved in the trial and respective contacts please click HERE.