We are honored and proud to be members of the Duchenne muscular dystrophy (DMD) community – one that is built on strength and determination. We strive to discover, develop and bring to patients therapies that will make a meaningful difference in the lives of those who are affected by this debilitating disease.
Clinical trials are designed to assess the safety and efficacy of potential new treatments and are an important part of the process of bringing these treatments to the community. We are grateful to the patients, families, and clinicians who participate, as each person’s contribution matters tremendously.
A word from our Chief Medical Officer, Dr. Joanne Donovan: "We understand that the decision to involve your child in a clinical trial is important and often difficult to make. We are continuously looking for ways to make participation in our trials easier on the whole family and are working with the community to make this possible. If you have suggestions or questions specific to our trials, please feel free to email me at email@example.com. If you are interested in learning more about edasalonexent and the MoveDMD® trial, you can sign up here.
Edasalonexent (formerly known as CAT-1004), is an oral investigational drug that inhibits NF-ĸB. In boys with DMD, the absence of dystrophin combined with mechanical stress in muscle leads to an activation of NF-ĸB. Activated NF-ĸB drives muscle damage and prevents muscle regeneration. Edasalonexent is an investigational drug that has not been approved by the US Food and Drug Administration. Edasalonexent is not a corticosteroid.
We are conducting a clinical trial of edasalonexent (CAT-1004) in boys ages 4 to 7 with DMD (any confirmed mutation). This trial is known as the MoveDMD trial – a clinical trial investigating the safety and efficacy of edasalonexent in DMD.
The first part of the MoveDMD trial (Part A) included 7 days of treatment with edasalonexent and the goal was to evaluate the safety, tolerability and pharmacokinetics of edasalonexent. Pharmacokinetics means how the drug moves through the body.
In Part B, we evaluated the safety and efficacy of edasalonexent in DMD (any confirmed mutation) and participants were randomized to take edasalonexent or placebo for a 12-week period. Thirty-one boys enrolled in Part B and all completed the trial. Parts A and B of the trial have been completed.
Following completion of the 12-week placebo-controlled Part B, patients continue on open-label edasalonexent for 36 weeks (Part C). We are monitoring safety as well as potential benefits of edasalonexent in Part C of the MoveDMD trial. We are assessing effects on the muscles of the lower and upper legs using magnetic resonance imaging (MRI), timed function tests, the North Star Ambulatory Assessment, muscle strength and a validated questionnaire is also being used.
We are no longer enrolling patients in the MoveDMD trial. Parts A and B of the MoveDMD trial are complete and the boys are continuing with the open-label extension (Part C).
The requirements for participating in the MoveDMD trial were:
The key inclusion and exclusion criteria and additional details about this clinical trial are available at https://clinicaltrials.gov/ct2/show/NCT02439216?term=cat-1004&rank=4.
Which centers are participating in the clinical trial?
For Parts A and B of the MoveDMD trial, costs for travel to the trial sites for participants and their immediate families were provided with support from Parent Project Muscular Dystrophy and the Muscular Dystrophy Association.
We have completed Phase 1 clinical trials with edasalonexent in adults. In these trials, edasalonexent consistently inhibited NF-ĸB and was well-tolerated. There were no safety concerns identified. The MoveDMD trial is a 3-part trial in boys affected by DMD. In Part A, edasalonexent was generally well tolerated, there were no safety issues, the pharmacokinetics were in line with what we expected and biomarker results demonstrated reduction in NF-ĸB activity.
We also reported top-line results for Part B of the MoveDMD trial, which evaluated the effects of edasalonexent using magnetic resonance imaging (MRI) T2 as the primary efficacy end point after 12 weeks of treatment. These data showed that the primary efficacy end point was not met. The 12-week time point was based on previously published data with steroids, and is earlier than typically used for trials with functional end points. We did observe some potential treatment-associated effects at 12 weeks in the 100 mg/kg/day treatment group in the timed function tests and North Star Ambulatory Assessment when compared to placebo although as expected the changes were not statistically significant. Edasalonexent was well-tolerated with no safety signals observed. The full analysis of data from Part B of the trial and from the ongoing open-label extension (Part C) is continuing and we plan to submit the data for presentation at upcoming scientific conferences. We will look to see if the signals from Part B strengthen in the longer-term data from Part C.