Rare Disease Report

Pfizer Completes Enrollment of Phase 2 Duchenne Trial

JUNE 01, 2017
James Radke
Pfizer announced it has completed patient enrollment in a Phase 2 clinical trial testing domagrozumab in boys with Duchenne muscular dystrophy (DMD).
A total of 121 DMD boys, aged 6 to 15 years of age, have enrolled.  The study will last 2 years and at the end of the trial, the boys may be eligible for an open label extension.
Domagrozumab is a monoclonal antibody directed towards myostatin, a protein in muscles that helps control muscle growth. It is hypothesized that by blocking myostatin there will be a decrease in the muscle wasting that occurs in boys with DMD.
Domagrozumab was granted Orphan Drug designation and Fast Track Designation by the FDA.
Pfizer has not stated when data from this trial will be made available.
Last year, we talked with Michael Binks, MD, Vice President for Rare Disease Clinical Research at Pfizer talks about domagrozumab and the Phase 2 study. Clips from that interview are shown below.

About Duchenne Muscular Dystrophy (DMD)

DMD is a progressive, degenerative muscle disorder caused by a lack of a functional dystrophin protein, a protein that helps keep muscle cells intact. Symptoms begin in early childhood and boys can lose the ability to walk as early as age 10. These patients, mostly boys, experience life-threatening heart and lung complications in their late teens and twenties.
There are many subsets of DMD based on where the mutation occurs on the dystrophin gene. Domagrozumab’s efficacy should be independent of the type of mutation and therefore equally effective in all DMD boys.

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