PolarisDMD: Phase 3 study of Edasalonexent (formerly CAT-1004) in people with Duchenne

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy

Questions and Answers

What stage is this research?

This will be a global Phase 3 trial in Duchenne muscular dystrophy to evaluate the efficacy and safety of edasalonexent. This trial is actively recruiting participants.

What is the goal or purpose of this research?

Edasalonexent is an oral small molecule investigational drug candidate that inhibits NF-kB, which is the key link between loss of dystrophin and disease pathology and which plays a fundamental role in the initiation and progression of skeletal and cardiac muscle disease in Duchenne. By inhibiting NF-kB, we believe that edasalonexent may reduce muscle damage and increase muscle regeneration in boys with Duchenne without the side effects of steroids.

Who is funding this study?

This research is being done by Catabasis, a clinical-stage biopharmaceutical company with a mission to bring hope and life-changing therapies to patients and their families.

Who is eligible to participate in this study?

This trial will enroll approximately 125 patients with the following criteria:

  • Age 4 to 7 (up to 8th birthday)
  • Able to perform stand from supine without assistance in ≤ 10 seconds and able to perform the 10MWT and 4-stair climb
  • Not on corticosteroids for at least 6 months
  • Not on other investigational therapies for at least 1 month, can be on stable eteplirsen

The key inclusion and exclusion criteria for this clinical trial will be available on www.clinicaltrials.gov.

What do I have to do if I decide to participate in this study?

The trial is a randomized, double-blind and placebo-controlled study with 2 boys receiving edasalonexent for every 1 boy receiving placebo. The drug is given as capsules which will be taken orally, three times per day.

Attend clinic visits every 3 months to assess North Star Ambulatory Assessment, time to stand, 4-stair climb, 10-meter walk/run, safety measures and assessments of growth, cardiac and bone health. Neither muscle biopsies nor MRI will be required for this study.

How long will this study last, and will I have access to the drug/treatment once the study has ended?

The anticipated length of the study is 12 months. After 12 months in the study, all boys are expected to receive edasalonexent in an open-label extension study.

Where does this study take place?

This study takes place at approximately 25 trial centers in the U.S. as well as sites in Canada, Europe, Israel and Australia.

How many visits to the study site are necessary?

  • 6 visits (screening, baseline and then every 3 months)

Can any visits be done locally?

  • With approximately 25 trial centers in the U.S., Catabasis will assist with matching participants to the closest trial center so travel burden is minimized. Visits to the trial center are every 3 months with a phone call between visits.

Is there any funding to help pay for travel?

  • Catabasis provides a travel program through a travel agency which is coordinated by the trial center.

Will I get paid for participating in this study?

Catabasis will provide study drug and all procedures and tests done specifically for this research study as well any expenses related to travel including lodging, meals and transportation.

Why should I consider participating in this study?

While no personal benefit can ever be guaranteed by participation in a clinical trial, you may learn more about DMD while you are in this study. The information that is collected from the study may help researchers find new treatments for future subjects. In the MoveDMD Phase 2 trial and open-label extension, slowing of disease progression was observed through 72 weeks of edasalonexent treatment compared to periods when subjects were off-treatment.

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