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 us at DMDtrials@catabasis.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 medicine that inhibits NF-ĸB and is a potential treatment for DMD. 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.
Phase 1 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 the 12-week Phase 2 of the MoveDMD trial (Part B), we evaluated the safety and efficacy of edasalonexent in DMD (any confirmed mutation) and participants were randomized to take edasalonexent or placebo. Thirty-one boys enrolled in and completed the 12-week Phase 2. The Phase 1 and the 12-week Phase 2 portions of the trial have been completed.
Following completion of the 12-week placebo-controlled Phase 2, patients are continuing on open-label edasalonexent (Part C).
We are no longer enrolling patients in the MoveDMD trial. The open-label extension is ongoing. 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?
Costs for travel to the trial sites for participants and their immediate families are provided with support from Parent Project Muscular Dystrophy and the Muscular Dystrophy Association.
In Phase 1 clinical trials with edasalonexent in adults, edasalonexent consistently inhibited NF-ĸB and was well-tolerated. There were no safety concerns identified.
In Phase 1 of the MoveDMD trial in boys affected by DMD, edasalonexent was well tolerated, there were no safety issues, the pharmacokinetics were in line with what we expected, and biomarker results demonstrated inhibition of NF-ĸB activity.
With 12 weeks of edasalonexent treatment in Phase 2 of the MoveDMD trial in boys affected by DMD, numerical improvements were observed in well-established and pre-specified functional assessments. These numerical improvements in the functional assessments demonstrated reductions in the rate of functional decline in both placebo-controlled and crossover analyses. The crossover analysis compared changes during an off-treatment period to edasalonexent treatment for boys who were in also in Phase 1 of the trial. Importantly, the functional assessments tested have precedence as endpoints in pivotal trials in DMD. The primary endpoint in the 12-week Phase 2, which was an exploratory MRI biomarker endpoint, was not met. Edasalonexent was well-tolerated with no safety signals observed.
In the open-label extension of the MoveDMD trial, edasalonexent substantially slowed DMD disease progression through 36 weeks of treatment. Across all key assessments of muscle function, consistent improvements were observed in the rate of decline after 24 and 36 weeks of oral 100 mg/kg/day edasalonexent treatment compared to the rate of change in the control period for boys prior to receiving edasalonexent treatment. The key assessments of muscle function were age-appropriate timed function tests (10-meter walk/run, 4-stair climb and time to stand) and the North Star Ambulatory Assessment (a composite endpoint evaluating physical function across 17 tests). Improvements were also seen across additional measures of muscle health. Edasalonexent continued to be well tolerated with no safety signals observed in the trial.
We are planning to start a single global Phase 3 trial in DMD in the first half of 2018 to evaluate the efficacy and safety of edasalonexent. We are not yet recruiting for this Phase 3 trial. The trial is anticipated to enroll approximately 125 patients ages 4 to 7 regardless of mutation type who have not been on steroids for at least 6 months. The trial is planned to be randomized, double-blind and placebo-controlled with 2 boys receiving edasalonexent for every 1 boy receiving placebo. The primary endpoint for the trial is anticipated to be change in North Star Ambulatory Assessment after 12 months of treatment with edasalonexent compared to placebo. After 12 months in the study, all boys are expected to receive edasalonexent in an open-label extension. We are in the process of identifying Phase 3 clinical trial sites and are exploring sites in North America, Europe and Australia. The list of recruiting sites will be shared closer to the start of the study.
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