NCT03652259TerminatedPhase 1
A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)
Sarepta Therapeutics, Inc.1 site in 1 country6 target enrollmentStarted: October 27, 2018Last updated:
Conditions
Interventions
Trial Snapshot
- Phase
- Phase 1
- Status
- Terminated
- Sponsor
- Sarepta Therapeutics, Inc.
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Study Overview
Brief Summary
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 Design
- Study Type
- Interventional
- Allocation
- Non Randomized
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 4 Years to 15 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Males or females of any ethnic group
- •β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles
- •Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs
- •A 100 meter walk/run (MWR) test result: ≥40 % of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit
Exclusion Criteria
- •Active viral infection based on clinical observations
- •Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction (LVEF) <40%
- •Serological evidence of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
- •Diagnosis of (or ongoing treatment for) an autoimmune disease
- •Abnormal laboratory values considered clinically significant
- •Concomitant illness or requirement for chronic drug treatment that, in the opinion of the Principal Investigator, creates unnecessary risks for gene transfer.
- •Other inclusion/exclusion criteria apply.
Arms & Interventions
Cohort 2: SRP-9003
Experimental
Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
Intervention: SRP-9003 (Genetic)
Cohort 1: SRP-9003
Experimental
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.
Intervention: SRP-9003 (Genetic)
Outcomes
Primary Outcomes
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Time Frame: Baseline up to 7 years
Secondary Outcomes
- Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers(Baseline, Day 60)
- Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence(Baseline, Day 60)
- Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot(Baseline, Day 60)
Investigators
Study Sites (1)
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