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

Background: Edasalonexent (CAT-1004) is an orally-administered novel small molecule drug designed to inhibit NF-κB and potentially reduce inflammation and fibrosis to improve muscle function and thereby slow disease progression and muscle decline in Duchenne muscular dystrophy (DMD). Objective: This...

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Veröffentlicht in:	Journal of neuromuscular diseases 2021-01, Vol.8 (5), p.769-784
Hauptverfasser:	Finkel, Richard S., McDonald, Craig M., Lee Sweeney, H., Finanger, Erika, Neil Knierbein, Erin, Wagner, Kathryn R., Mathews, Katherine D., Marks, Warren, Statland, Jeffrey, Nance, Jessica, McMillan, Hugh J., McCullagh, Gary, Tian, Cuixia, Ryan, Monique M., O’Rourke, Declan, Müller-Felber, Wolfgang, Tulinius, Mar, Burnette, W. Bryan, Nguyen, Cam-Tu, Vijayakumar, Kayal, Johannsen, Jessika, Phan, Han C., Eagle, Michelle, MacDougall, James, Mancini, Maria, Donovan, Joanne M.
Format:	Artikel
Sprache:	eng
Schlagworte:	Adverse events Diarrhea Double-blind studies Duchenne's muscular dystrophy Dystrophin Fibrosis Muscular dystrophy NF-κB protein Patients Pediatrics Placebos Research Report Statistical analysis
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Zusammenfassung:	Background: Edasalonexent (CAT-1004) is an orally-administered novel small molecule drug designed to inhibit NF-κB and potentially reduce inflammation and fibrosis to improve muscle function and thereby slow disease progression and muscle decline in Duchenne muscular dystrophy (DMD). Objective: This international, randomized 2 : 1, placebo-controlled, phase 3 study in patients ≥4 –
ISSN:	2214-3599 2214-3602
DOI:	10.3233/JND-210689