Clinical Trials Spotlight - Duchenne Muscular Dystrophy

The Children’s CTSU is currently supporting a Phase III trial studying the drug Edasalonexent (Edasa) in pediatric patients with Duchenne Muscular Dystrophy (DMD). Sponsored by Catabasis and lead by Principal Investigator Erin Neil Knierbein, DO, and Children's CTSU Study Coordinator Amy Hurst, this randomized, double-blind, and placebo-controlled study is attempting to determine the effectiveness of Edasa in reducing the symptoms of DMD and its ability to slow down the disease progression.

DMD is a rare, progressively debilitating, and ultimately fatal neuromuscular genetic disorder impacting 1 in every 3,500 to 5,000 males worldwide. DMD is caused by mutations in the gene that encodes dystrophin; a protein that acts as a shock absorber and protects muscles from injury. Without dystrophin, stress placed on membranes during muscle contraction causes them to tear. This degeneration of muscle fibers attracts inflammatory cells and results in the replacement of healthy muscle with fatty and fibrosis tissue. Because of this, affected children are typically wheelchair-dependent by the age of 14.

Since there is no cure, current standard of care for DMD includes administration of glucocorticoids, which can delay some of the ailments of the disease (such as the onset of scoliosis). However, they do not halt disease progression and are also limited by numerous side effects. By inhibiting NF-kB, which is a key link between the loss of dystrophin and disease pathology, Edasa has the potential to provide benefits to patients beyond those afforded by glucocorticoids and without their negative side effects.

Amy Hurst shared, “The University of Michigan has become the top recruitment site for the Edasalonexent trials. Thus far, Edasa has shown great promise in slowing down the progression of the disease.”

To learn more about the Children’s CTSU Edasa trial click here.