Researchers at Catabasis are looking for boys with Duchenne muscular dystrophy (DMD) to participate in a Phase 3 study (PolarisDMD). The goal of the study is to evaluate the safety and efficacy of edasalonexent, which researchers believe may slow down progression of skeletal and cardiac muscle disease in DMD by inhibiting NF-kB. NF-kB is a protein complex that helps to activate the immune system, which leads to the inflammation associated with muscle weakness and disease progression.
Participants will be randomized to receive either edasalonexent or placebo (two participants receiving edasalonexent for every one receiving placebo). At the end of the trial, an open-label extension will offer edasalonexent to those who received placebo if the safety profile is acceptable.
Total study duration for each patient will be about one year, with clinic visits every three months. At each visit, efficacy will be evaluated by the performance of the North Star Ambulatory Assessment, timed function tests and cardiac and bone assessments. Throughout the study, participants will continue to see their regular doctor for routine care.
In order to be eligible to participate, boys must:
- Be between the ages of 4 and 7 (up to 8th birthday)
- Be ambulatory
- Have not used corticosteroids for 24 weeks prior to the study’s start
- Have a confirmed genetic diagnosis of DMD
- Meet additional study criteria
Travel support for each visit is available.
To find out more information about the study, including a list of active trial sites, you may visit https://clinicaltrials.gov/ct2/show/NCT03703882 or the Catabasis website at https://www.catabasis.com/patients-families/polaris-dmd-clinical-trial.php
To inquire about participation, contact Maria C. Mancini at DMDtrials@catabasis.com