This is an open-label gene transfer therapy study evaluating the safety of and expression from delandistrogene moxeparvovec in participants with DMD. The maximum participant duration for this study is 156 weeks.
Enrollment for Cohorts 1 through 7 has been completed. Cohort 8 is currently enrolling new participants.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
83
Single IV infusion of delandistrogene moxeparvovec
Arkansas Children's Hospital
Little Rock, Arkansas, United States
RECRUITINGStanford University
Palo Alto, California, United States
RECRUITINGUniversity of California, Davis
Sacramento, California, United States
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot
Time frame: Baseline, Week 12
Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot
Time frame: Week 12
Cohort 8: Number of Participants with Acute Liver Injury (ALI)
Time frame: Baseline up to Week 72
Vector Shedding, Measured in Urine, Saliva, and Stool Samples Post-Infusion
Time frame: Day 1 up to Week 104
Level of Antibody Titers to Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74)
Time frame: Day 2 up to Week 156
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs)
Time frame: Baseline up to Week 156
Cohort 8: Number of Participants With Infections, Edema, Wound-healing Complications, Hyperlipidemia, Angioedema, and Intestinal Lung Disease/ Non-infectious Pneumonitis
Time frame: Baseline up to Week 72
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by Immunofluorescence (IF) Fiber Intensity
Time frame: Baseline, Week 12
Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12, as Measured by IF Percent Dystrophin Positive Fibers (PDPF)
Sarepta Therapeutics Inc., For Clinical Trial Information, Select Option 4
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Washington University in St. Louis
St Louis, Missouri, United States
RECRUITINGNationwide Children's Hospital
Columbus, Ohio, United States
ACTIVE_NOT_RECRUITINGNeurology Rare Disease Center
Flower Mound, Texas, United States
RECRUITINGChildren's Hospital of The King's Daughters
Norfolk, Virginia, United States
RECRUITINGTime frame: Baseline, Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF Fiber Intensity
Time frame: Week 12
Quantity of Delandistrogene Moxeparvovec Dystrophin Protein Expression at Week 12 as Measured by IF PDPF
Time frame: Week 12
Cohort 8: Number of Participants With Hepatic AEs, Hepatic Biomarkers, and Laboratory Assessments Indicative of Either Acute Hepatocellular Injury or Acute Liver Dysfunction
Time frame: Baseline up to Week 72
Cohort 8: Number of Participants with Severe ALI
Time frame: Baseline up to Week 72
Cohort 8: Duration of Steroids Administered
Time frame: Baseline up to Week 72