The primary objective of this study is to evaluate the safety of SRP-9004.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
4
Intravenous (IV) infusion.
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Number of Participants with Treatment-emergent Adverse Events (TEAEs), Adverse Events of Special Interest (AESI) and Treatment-emergent Serious Adverse Events (SAEs)
Time frame: Up to 60 months
Change from Baseline Through Day 60 in Quantity of Alpha-sarcoglycan (α-SG) Protein Expression
Time frame: Baseline, Day 60
Change from Baseline Through Month 24 in Quantity of α-SG Protein Expression
Time frame: Baseline, Month 24
Change from Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Performance of the Upper Limb (PUL) Version 2.0 Total Score
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Time to Rise From Floor
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Time to Ascend 4 Steps
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Time to Complete 10-meter Walk/Run (10MWR)
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Time to Complete 100-meter Walk/Run (100MWR)
Time frame: Baseline, Month 60
Change from Baseline through Month 60 in Creatine Kinase (CK) Level
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Time frame: Baseline, Month 60
Time to Change of Disease Milestones (Loss of Ambulation)
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Skeletal Muscle Magnetic Resonance Imagining (MRI) Findings
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Wearable Device Stride Velocity 95% (SV95C) (Ambulatory Participants Only)
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Vital Capacity (FVC)
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FVC Percentage (%) Predicted
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Forced Expiratory Volume in 1 Second (FEV1)
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by FEV1 % Predicted
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Peak Expiratory Flow Rate (PEFR)
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Inspiratory Pressure (MIP)
Time frame: Baseline, Month 60
Change from Baseline Through Month 60 in Pulmonary Functions as Assessed by Maximal Expiratory Pressure (MEP)
Time frame: Baseline, Month 60