AOC 1044 for Duchenne Muscular Dystrophy
(SAFARI44 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
Are You a Good Fit for This Trial?
This trial is for boys aged 7 to 16 who can walk and have Duchenne muscular dystrophy (DMD) caused by a specific gene mutation that can be treated with exon 44 skipping. They must have been on stable steroid treatment for at least six months.Inclusion Criteria
What Are the Treatments Tested in This Trial?
Interventions
- AOC 1044
Trial Overview
The study tests AOC 1044, an intravenous medication, against a placebo in boys with DMD eligible for exon 44 skipping. Participants are randomly assigned to receive either the drug or placebo without knowing which one they get (double-blind).
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Placebo Group
Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W
Participants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W
Find a Clinic Near You
Who Is Running the Clinical Trial?
Avidity Biosciences, Inc.
Lead Sponsor
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