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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2024
• Main ID: NCT03652259
• Date of registration: 28/08/2018
• Prospective Registration: Yes
• Primary sponsor: Sarepta Therapeutics, Inc.
• Public title: Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
• Scientific 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 (ß-Sarcoglycan Deficiency)
• Date of first enrolment: October 27, 2018
• Target sample size: 6
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT03652259
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Medical Director
• Affiliation: Sarepta Therapeutics, Inc.

Key inclusion & exclusion criteria

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.



Age minimum: 4 Years
Age maximum: 15 Years
Gender: All

Health Condition(s) or Problem(s) studied

Limb-Girdle Muscular Dystrophy, Type 2E

Intervention(s)

Genetic: SRP-9003

Primary Outcome(s)

Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) [Time Frame: Baseline up to 7 years]

Secondary Outcome(s)

Change From Baseline in Quantity of ß-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers [Time Frame: Baseline, Day 60]

Change From Baseline in Quantity of Beta-Sarcoglycan (ß-SG) Protein Expression at Day 60, as Measured by Western Blot [Time Frame: Baseline, Day 60]

Change From Baseline in Quantity of ß-SG Protein Expression at Day 60, as Measured by Immunofluorescence [Time Frame: Baseline, Day 60]

Secondary ID(s)

IRB17-00253

SRP-9003-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors