AOC 1044: AOC 1044 will be administered via intravenous (IV) infusion
Study 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.
Eligibility
Sex
MALE
Min age
7 Years
Max age
27 Years
Healthy volunteers
No
Key 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
* Weight ≥ 23 kg
* Ambulatory or non-ambulatory
* Ambulatory participants: LVEF ≥50% and FVC≥50%
* Non-ambulatory participants: LVEF ≥45% and FVC≥40%
* PUL 2.0 entry item A ≥3
* If on corticosteroids, stable dose for 30 days before screening and throughout the study
Key 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
Primary outcome measure(s)
Incidence of Treatment Emergent Adverse Events (TEAEs) — Through study completion (approximately 2 years)
Trial sites (10)
Facility
City
Region
Status
Arkansas Children&#39;s Hospital
Little Rock
Arkansas
University of California, San Diego, Rady's Children's Hospital
La Jolla
California
UC Davis Medical Center
Sacramento
California
Lucille Packard Children&#39;s Hospital at Stanford
San Carlos
California
Rare Disease Research - Atlanta
Atlanta
Georgia
University of Massachusetts Medical School
Worcester
Massachusetts
Gillette Children's Specialty Healthcare
Saint Paul
Minnesota
Rare Disease Research
Hillsborough
North Carolina
Abigail Wexner Research Institute at Nationwide Children's Hospital
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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