Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

Limb-Girdle Muscular Dystrophy, Type 2E Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03652259
• Other study ID #: SRP-9003-101
• Secondary ID: IRB17-00253
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: October 27, 2018
• Est. completion date: February 14, 2027

Study information

• Verified date: February 2024
• Source: Sarepta Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 6
• Est. completion date: February 14, 2027
• Est. primary completion date: February 14, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 15 Years

Eligibility

INCLUSION CRITERIA

• Males or females of any ethnic group
• ß-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.

Related Conditions & MeSH terms

• Limb-Girdle Muscular Dystrophy, Type 2E
• Muscular Dystrophies

Intervention

Genetic:
• SRP-9003
SRP-9003 will be administered through a single systemic injection.

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)		Baseline up to 7 years
Secondary	Change From Baseline in Quantity of Beta-Sarcoglycan (ß-SG) Protein Expression at Day 60, as Measured by Western Blot	ß-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.	Baseline, Day 60
Secondary	Change From Baseline in Quantity of ß-SG Protein Expression at Day 60, as Measured by Immunofluorescence	ß-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.	Baseline, Day 60
Secondary	Change From Baseline in Quantity of ß-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers		Baseline, Day 60