AOC 1044 Exon 44 Skipping Therapy for Duchenne Muscular Dystrophy: EXPLORE44 Clinical Trial

Active, Not RecruitingPhase 2Exon Skipping

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

If on corticosteroids, stable dose for 30 days before screening and throughout the study.

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

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

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Study Requirements and Criteria