AOC 1044 for Duchenne Muscular Dystrophy
(EXPLORE44 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.
Who Is on the Research Team?
Carmen Castrillo, MD
Principal Investigator
Avidity Biosciences, Inc.
Are You a Good Fit for This Trial?
This trial is for healthy adults and those with Duchenne Muscular Dystrophy (DMD) who can benefit from exon 44 skipping. Participants must be aged 18-55 years with a BMI of 18.5 to 32 kg/m2.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part A
Single dose administration of AOC 1044 or placebo in healthy adult volunteers
Treatment Part B
Multiple ascending dose administration of AOC 1044 or placebo in participants with DMD
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- AOC 1044
Find a Clinic Near You
Who Is Running the Clinical Trial?
Avidity Biosciences, Inc.
Lead Sponsor