The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
6
SRP-9003 will be administered through a single systemic injection.
Nationwide Children's Hospital
Columbus, Ohio, United States
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Time frame: Baseline up to 7 years
Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot
β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.
Time frame: Baseline, Day 60
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence
β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.
Time frame: Baseline, Day 60
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers
Time frame: Baseline, Day 60
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