Status and phase
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About
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.
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Part A:
Key Inclusion Criteria:
Key Exclusion Criteria:
Part B:
Key Inclusion Criteria:
Aged 7 to 27 years, inclusive, at the time of informed consent
Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
Confirmation of DMD gene mutation amenable to exon 44 skipping
Weight ≥ 23 kg
Ambulatory or non-ambulatory
PUL 2.0 entry item A ≥3
If on corticosteroids, stable dose for 30 days before screening and throughout the study
Key Exclusion Criteria:
Primary purpose
Allocation
Interventional model
Masking
64 participants in 4 patient groups, including a placebo group
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Central trial contact
Avidity Biosciences, Inc.
Data sourced from clinicaltrials.gov
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