NCT03652259

Brief 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.

Trial Health

57
Monitor

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
Completed

Started Oct 2018

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
terminated

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

First Submitted

Initial submission to the registry

August 28, 2018

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 29, 2018

Completed
2 months until next milestone

Study Start

First participant enrolled

October 27, 2018

Completed
6.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 14, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 14, 2025

Completed
Last Updated

January 20, 2026

Status Verified

January 1, 2026

Enrollment Period

6.2 years

First QC Date

August 28, 2018

Last Update Submit

January 12, 2026

Conditions

Limb-Girdle Muscular Dystrophy, Type 2E

Keywords

limb girdle muscular dystrophyLGMD2Ebeta-sarcoglycangene transferadeno-associated virusLGMDR4

Outcome Measures

Primary Outcomes (1)

  • Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)

    Baseline up to 7 years

Secondary Outcomes (3)

  • Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot

    Baseline, Day 60

  • Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence

    Baseline, Day 60

  • Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers

    Baseline, Day 60

Study Arms (2)

Cohort 1: SRP-9003

EXPERIMENTAL

Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.

Genetic: SRP-9003

Cohort 2: SRP-9003

EXPERIMENTAL

Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.

Genetic: SRP-9003

Interventions

SRP-9003GENETIC

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

Also known as: LGMD2E vector, bidridistrogene xeboparvovec
Cohort 1: SRP-9003Cohort 2: SRP-9003

Eligibility Criteria

Age4 Years - 15 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Males or females of any ethnic group
  • β-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

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Related Publications (1)

  • Mendell JR, Pozsgai ER, Lewis S, Griffin DA, Lowes LP, Alfano LN, Lehman KJ, Church K, Reash NF, Iammarino MA, Sabo B, Potter R, Neuhaus S, Li X, Stevenson H, Rodino-Klapac LR. Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results. Nat Med. 2024 Jan;30(1):199-206. doi: 10.1038/s41591-023-02730-9. Epub 2024 Jan 4.

    PMID: 38177855DERIVED

MeSH Terms

Conditions

Limb-girdle muscular dystrophy, type 2EMuscular 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
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 28, 2018

First Posted

August 29, 2018

Study Start

October 27, 2018

Primary Completion

January 14, 2025

Study Completion

January 14, 2025

Last Updated

January 20, 2026

Record last verified: 2026-01

Locations

US(1)