AOC 1044 Exon 44 Skipping Therapy for Duchenne Muscular Dystrophy: EXPLORE44 Clinical Trial
Active, Not RecruitingPhase 2Exon Skipping
Study Overview
- Age
- 7–27 years
- Phase
- Phase 2
- Sponsor
- Avidity Biosciences
- Therapeutic Approach
- Exon Skipping
- Variant Requirement
- 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.
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Study Start (Actual)
- 2024-01-22
- Primary Completion (Estimated)
- 2027-04-30
- Study Completion (Estimated)
- 2027-07-31
- Enrollment (Estimated)
- 39
- Countries
- United States
Study Requirements and Criteria
Steroid Use
If on corticosteroids, stable dose for 30 days before screening and throughout the study.
Inclusion Criteria
- Rollover Participants:
- Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor
- No significant tolerability issues with AOC 1044
- De novo Participants:
- 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
- PUL 2.0 entry item A ≥3
Exclusion Criteria
- Rollover Participants:
- Presence of any new condition or worsening of existing condition that could affect a participant's safety or ability to comply with study procedures
- De novo Participants:
- Serum hemoglobin < lower limit of normal
- Uncontrolled hypertension or diabetes
- Prior treatment with any cell or gene therapy
- Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent
- Recently treated with an investigational drug
- History of multiple drug allergies
Clinical Trial Registry
NCT ID
NCT06244082This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.