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A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids
The purpose of the study is to assess the efficacy of idebenone in delaying the loss of respiratory function in patients with DMD receiving concomitant glucocorticoid steroids
sponsor: Santhera Pharmaceuticals
location: United States, Austria, Belgium, Bulgaria, France, Germany, Ireland, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom
A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD patients by measuring biological and clinical endpoints in two parts: a 48-week randomized, double-blinded, placebo-controlled period (Part 1), and a 96-week, double-blinded extension period (Part 2).
sponsor: Sarepta Therapeutics
location: United States
A safety and efficacy study of ATL1102 in patients with Duchenne muscular dystrophy
purpose: Current DMD therapies are aimed at increasing dystrophin levels and reducing inflammation. Improved anti-inflammatory therapies are needed to safely treat this pathology and delay disease progression. This study will be conducted in a single-centre and assess the safety, efficacy and PK of ATL1102 in non-ambulatory patients with DMD. Read more:...
sponsor: Antisense Therapeutics Limited
A Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy (PolarisDMD)
purpose: The PolarisDMD study is a Phase 3, global study to evaluate the efficacy and safety of edasalonexent in pediatric patients with a genetically confirmed diagnosis of DMD. Male patients from 4-7 years of age (up to 8th birthday) will be enrolled. Edasalonexent is an orally administered small molecule that inhibits NF-kB, which is the key link between loss of dystrophin and disease pathology and plays a fundamental role in the initiation an...
sponsor: Catabasis Pharmaceuticals
location: United States, Australia, Canada
A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy
This is a multicenter, double blind, placebo controlled, multiple dose study to examine the safety and efficacy of MNK-1411 in male subjects 4 to 8 years of age (inclusive) with Duchenne Muscular Dystrophy (DMD). Research: Anti-inflammatory
location: United States, Israel, Mexico, Spain, Turkey
A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD)
purpose: Brief Summary: This Phase IIb study is a randomized, double-blind, parallel group, placebo and active-controlled study to evaluate the efficacy, safety, PD, and population PK of vamorolone administered orally at daily doses of 2.0 mg/kg and 6.0 mg/kg versus prednisone 0.75 mg/kg/day and placebo over a Treatment Period of 24 weeks, and to evaluate persistence of effect over a Treatment Period of 48 weeks in ambulant boys ages 4 to <7 years w...
sponsor: ReveraGen BioPharma, Inc.
location: United States, Australia, Canada, Israel, Sweden, United Kingdom