According to a story from drugs.com, the biopharmaceutical company Catabasis pharmaceuticals recently provided updates on the status of its Phase 3 clinical trial testing its investigational therapy CAT-1004, also known as edasalonexent. This product is in development as a treatment for Duchenne muscular dystrophy, a rare degenerative disorder. This trial is expected to include trial sites around the world.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, boys are mostly affected, with girls only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.
As if January 2rd, 2019, the clinical trial sites in the US are actively enrolling Duchenne muscular dystrophy patients to participate. There are currently nine sites taking part in the process, with a further ten slated to begin similar operations in the coming weeks. Catabasis is also in the midst of submitting international clinical trials applications. The company has so far received approvals in a number of European countries and in Canada. The first sites are expected to open up in Canada by the end of the month. The company ultimately hopes to have trial sites in Australia and Israel also in the next few months. These sites are expected to eventually number 40 in total.
This trial is expected to last for one year and will include 125 participating patients between the ages of four and seven. The data from this trial is intended to support an eventual application for public approval and will hopefully reinforce the encouraging results seen in earlier trials of CAT-1004.