Clinical Trials in the USA / NCT05876780
Active, not recruiting
Phase 1
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 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Limb Girdle Muscular Dystrophy
Investigational drug(s) / intervention(s)
SRP-9003
SRP-9003: Single IV infusion of SRP-9003
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.
Eligibility
Inclusion Criteria:
* Cohort 1 only: Ambulatory per protocol specified criteria. - Cohort 2 only: Non-ambulatory per protocol specified criteria and 4 to 50 years of age.
* Possesses 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-sarcoglycan gene Deoxyribonucleic acid (DNA) (SGCB) gene mutations based on documented clinical findings.
* Ability to cooperate with muscle testing.
Exclusion Criteria:
* Presence of any other clinically significant illness or medical condition, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, neuromuscular (other than LGMD2E/R4), or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.
* Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
* Any contraindication to use of corticosteroid.
Note: Other inclusion or exclusion criteria could apply.
Primary outcome measure(s)
- Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) — Baseline up to Month 60
- Change from Baseline in β-SG Protein Expression Quantity Assessed by Immunofluorescence (IF) Fiber Intensity at Day 60 — Baseline, Day 60
- Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Day 60 — Baseline, Day 60
- Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Day 60 — Baseline, Day 60
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| Nationwide Children's Hospital | Columbus | Ohio | |
| St. Jude Children's Research Hospital | Memphis | Tennessee |
More Sarepta Therapeutics, Inc. trials in the USA
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
Active, not recruiting
A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study
Enrolling by invitation
Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05876780 on ClinicalTrials.gov ↗ ← All trials in the USA