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.
AOC 1044-CS2 (EXPLORE44-OLE) is an open label, extension study to Part B of AOC 1044-CS1 (EXPLORE44). AOC 1044-CS2 is designed to evaluate the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044. All participants who enroll in AOC 1044-CS2 will receive active treatment. The treatment period is 2 years with IV dosing every 6 weeks. Once participants have completed active treatment, they will be followed through a 3-month safety follow-up period. The sponsor may extend active treatment beyond 2 years at a future timepoint.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
39
AOC 1044 will be administered via intravenous (IV) infusion
Arkansas Children's Hospital
Little Rock, Arkansas, United States
University of California, San Diego, Rady's Children's Hospital
La Jolla, California, United States
UC Davis Medical Center
Sacramento, California, United States
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: Through study completion (approximately 2 years)
Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102
Time frame: Through study completion (approximately 2 years)
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Lucille Packard Children's Hospital at Stanford
San Carlos, California, United States
Rare Disease Research - Atlanta
Atlanta, Georgia, United States
University of Massachusetts Medical School
Worcester, Massachusetts, United States
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, United States
Rare Disease Research
Hillsborough, North Carolina, United States
Abigail Wexner Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States
Neurology Rare Disease Center
Denton, Texas, United States