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A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

NCT05876780 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-02-19

No results posted yet for this study

Summary

The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.

Conditions

  • Limb Girdle Muscular Dystrophy

Interventions

GENETIC

SRP-9003

Single IV infusion of SRP-9003

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-12-19
Primary Completion
2028-08-28
Completion
2028-08-28
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05876780 on ClinicalTrials.gov