Pfizer Currently Recruiting Participants For Phase 2 Duchenne Muscular Dystrophy Clinical Trial

Pfizer Currently Recruiting Participants For Phase 2 Duchenne Muscular Dystrophy Clinical Trial

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.

Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
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Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
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