This is a gene transfer therapy study evaluating the safety of and delandistrogene moxeparvovec dystrophin protein expression from delandistrogene moxeparvovec following therapeutic plasma exchange (plasmapheresis) in ambulatory male participants with DMD and pre-existing antibodies to AAVrh74 over a period of 59 weeks.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
Single IV infusion of delandistrogene moxeparvovec
Therapeutic plasma exchange procedure
University of Florida, College of Medicine
Gainesville, Florida, United States
Washington University School of Medicine in St. Louis
St Louis, Missouri, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression Adjusted by Muscle Content Biopsied Muscle as Measured by Western Blot
Time frame: Baseline, Week 12
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by Immunofluorescence (IF) Fiber Intensity
Time frame: Baseline, Week 12
Change From Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression in Biopsied Muscle as Measured by IF Percent Dystrophin-positive Fibers (PDPF)
Time frame: Baseline, Week 12
Mean Concentration of Vector Genome Copies Using Polymerase Chain Reaction in Muscle Tissue Biopsy, After Delandistrogene Moxeparvovec Administration
Time frame: Week 12
Number of Participants with a Treatment Emergent Adverse Event (TEAE), Adverse Event of Special Interest (AESI), and Serious Adverse Event (SAE)
Time frame: Baseline up to End of Study (Up to Week 59)
Change from Baseline in rAAVrh74 Antibody Titers
Time frame: Baseline, Week 1
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