Clinical Trials

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.comIf you are interested in learning more about edasalonexent and the MoveDMD® trial, you can sign up here.

MoveDMD Trial

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.

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. Phase 1 and the 12-week Phase 2 have been completed.

Following completion of the 12-week placebo-controlled Phase 2, patients continue on open-label edasalonexent for 60 weeks (Part C). We are monitoring safety as well as potential benefits of edasalonexent in the open-label extension of the MoveDMD trial. We are assessing effects on the muscles of the lower and upper legs using magnetic resonance imaging (MRI) and on function using timed function tests, the North Star Ambulatory Assessment, muscle strength and a validated questionnaire (the Pediatric Outcomes Data Collection Instrument).

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:

  • Boys with a diagnosis of DMD (any confirmed mutation) between the ages 4 and 7 years
  • Ability to walk independently
  • No corticosteroid use within the past 6 months and no plans to start corticosteroids in the next 6 months

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?

  • University of Florida, Gainesville FL
  • Shriners Hospital for Children, Portland OR
  • Children's Hospital of Philadelphia (CHOP), Philadelphia PA
  • Nemours Children’s Hospital, Orlando FL
  • University of California, Los Angeles CA

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. 

 

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 generally 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, functional assessments 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. Edasalonexent is currently in the open-label extension of the MoveDMD trial with results expected in Q3 2017. We anticipate announcing plans for a Phase 3 trial in the second half of 2017.