Phase 3 PolarisDMD Trial [TERMINATED]

Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)

Hub Summary

 PolarisDMD is a global, placebo controlled, Phase 3 trial for edasolonexent (CAT-1004). Edasalonexent is an NF-kB inhibitor, which could provide an alternative to steroids. Edasalonexent has been shown to preserve muscle function and substantially slow Duchenne disease progression in the MoveDMD trial.

This trial will evaluate the efficacy and safety of edasolonexent in patients with DMD, and is intended to support an application for commercial licencing of edasolonexent.

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Study Number: NCT03703882

Description by Catabasis Pharmaceuticals

The PolarisDMD study is a global Phase 3 study to evaluate the efficacy and safety of edasalonexent (CAT-1004) in boys 4 to 7 years old affected by DMD.

Two boys will receive edasalonexent for each boy that receives placebo and after 12 months, all boys are expected to receive edasalonexent in an open-label extension. Edasalonexent is an oral therapy.

Edasalonexent is a potential foundational therapy that is being developed for all patients affected by DMD. Edasalonexent inhibits NF-kB, which drives inflammation, fibrosis and muscle degeneration and suppresses muscle regeneration.

At this time, the UK sites are at capacity however if you are interested in trial participation, please contact DMDtrials@catabasis.com

 
Primary Outcome Measures

• Change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo

 
Secondary Outcome Measures

• Timed function tests time to stand, 4-stair climb and 10-meter walk/run

• Additional assessments of growth, cardiac and bone health

 
Can I take part?

 
Inclusion Criteria

 
Exclusion Criteria