Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
NCT06244082 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 39
Last updated 2025-05-14
Summary
AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
Conditions
- DMD
- Duchenne Muscular Dystrophy
- Duchenne
- Exon 44
Interventions
- DRUG
-
AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Sponsors & Collaborators
-
Avidity Biosciences, Inc.
lead INDUSTRY
Principal Investigators
-
Carmen Castrillo, MD · Avidity Biosciences, Inc.
Study Design
- Allocation
- NA
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 7 Years
- Max Age
- 27 Years
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2024-01-22
- Primary Completion
- 2027-04-30
- Completion
- 2027-07-31
- FDA Drug
- Yes
Countries
- United States
Study Locations
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