NCT05876780

Brief 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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
28mo left

Started Dec 2022

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress59%
Dec 2022Aug 2028

Study Start

First participant enrolled

December 19, 2022

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

May 17, 2023

Completed
8 days until next milestone

First Posted

Study publicly available on registry

May 25, 2023

Completed
5.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 28, 2028

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 28, 2028

Last Updated

February 19, 2026

Status Verified

February 1, 2026

Enrollment Period

5.7 years

First QC Date

May 17, 2023

Last Update Submit

February 17, 2026

Conditions

Limb Girdle Muscular Dystrophy

Keywords

AmbulatoryNon-ambulatoryGene-DeliveryLGMD2E/R4β-SG deficiencyLGMDPerformance of Upper Limb (PUL)

Outcome Measures

Primary Outcomes (4)

  • 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

Secondary Outcomes (14)

  • Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Fiber Intensity at Month 24

    Baseline, Month 24

  • Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Month 24

    Baseline, Month 24

  • Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Month 24

    Baseline, Month 24

  • Change from Baseline in North Star Assessment for Dysferlinopathy (NSAD) at Month 60

    Baseline, Month 60

  • Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 meter Walk/Run, and the Timed Stair Ascend 4 Steps Test

    Baseline, Month 60

  • +9 more secondary outcomes

Study Arms (1)

SRP-9003

EXPERIMENTAL

Participants will receive single IV infusion of SRP-9003 on Day 1.

Genetic: SRP-9003

Interventions

SRP-9003GENETIC

Single IV infusion of SRP-9003

Also known as: scAAVrh74.MHCK7.hSGCB
SRP-9003

Eligibility Criteria

Age4 Years - 50 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies, Limb-Girdle

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Medical Director

    Sarepta Therapeutics, Inc.

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 17, 2023

First Posted

May 25, 2023

Study Start

December 19, 2022

Primary Completion (Estimated)

August 28, 2028

Study Completion (Estimated)

August 28, 2028

Last Updated

February 19, 2026

Record last verified: 2026-02

Locations

US(2)