Two Clinical Studies of an Experimental Drug for Duchenne Muscular Dystrophy Have Been Stopped

According to a recent press release, Pfizer Inc. has terminated two on-going clinical trials that were investigating domagrozumab (PF-06252616) as a potential treatment for Duchenne muscular dystrophy. This decision follows disappointing results that didn’t show a significant treatment effect, and is not due to safety reasons. For more detailed information about this, you can view the source press release here, at Pfizer’s website.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a genetic disorder that is characterised by progressive muscle weakness and degeneration. It is caused by genetic alterations to a gene that codes for the protein dystrophin. As a result, the body does not produce enough working dystrophin. Dystrophin is crucial for healthy muscle growth and maintenance, and the lack of this protein causes many of the symptoms associated with DMD.

About the Studies

The two studies that have been terminated were a Phase 2 study investigating the safety and efficacy of domagrozumab, and an open-label extension study. The Phase 2 study was a double-blind trial that took place over several centres. It compared the effects of the drug domagrozumab to a placebo in 121 boys with DMD who were aged between six and fifteen. In addition, all of the boys were given background corticosteroid therapy.

The Results

Originally, the Phase 2 trial was designed to take place over two years. However, analysis after one year of treatment showed that the study had not met its main endpoint – a difference between the placebo and domagrozumab groups in the mean change from baseline in a 4 stair climb test. Several other measurements failed to show an effect from treatment with domagrozumab. In the press release, Pfizer say that the decision to terminate the study came after a “thorough review” of the data, and that the company will continue to review the data to better understand the study. A spokesperson for Pfizer’s Rare Disease Research Unit said the company is “disappointed by these results”, but “extremely grateful” to all the boys and families who took part.

Pfizer says that it will continue to research DMD and other rare neuromuscular diseases, including in an on-going clinical trial of an investigational gene therapy for DMD (PF-06939926).

For more information about Pfizer’s decision, you can view the original press release by clicking here.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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