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NCT Number: NCT06246513

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

This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).

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Key information

Conditions

Limb-girdle Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophies, Limb-Girdle Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

4 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

University Hospital Leuven (UZ Leuven), Leuven, Vlaams Brabant, Belgium

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Cohort 1, only ambulatory participants:
  • Able to walk without assistive aid
  • 10MWR <30 seconds
  • NSAD ≥25
  • Cohort 2, only non-ambulatory participants:
  • 10MWR ≥30 seconds or unable to perform
  • PUL 2.0 entry scale score ≥3
  • Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
  • Able to cooperate with muscle testing
  • Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers <1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.

Exclusion criteria

  • Left ventricular ejection fraction < 40% or clinical signs and/or symptoms of cardiomyopathy
  • Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
  • Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
  • Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)

Other inclusion/exclusion criteria apply.

Treatment and study plan

SRP-9003

Biological

Solution for single IV infusion

Other names: scAAVrh74.MHCK7.hSGCB, bidridistrogene xeboparvovec

glucocorticoid

Drug

Oral tablet (prophylactic)

Other names: Prednisone or equivalent

Primary outcomes

  1. Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers

    Time frame: Baseline, Day 60

Secondary outcomes

  1. Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by Western Assay

    Time frame: Baseline, Day 60

  2. Cohort 1 and Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Intensity (PFI)

    Time frame: Baseline, Day 60

  3. Cohort 2: Change From Baseline in β-SG Expression at Day 60 Post-dose as Measured by IF Percent β-SG Positive Fibers (PβSGPF)

    Time frame: Baseline, Day 60

  4. Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in North Star Assessment for Limb-girdle Muscular Dystrophies (NSAD) Total Score

    Time frame: Baseline through Month 60

  5. Cohort 1 and Cohort 2: Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score

    Time frame: Baseline through Month 60

  6. Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test

    Time frame: Baseline through Month 60

  7. Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test

    Time frame: Baseline through Month 60

  8. Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test

    Time frame: Baseline through Month 60

  9. Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test

    Time frame: Baseline through Month 60

  10. Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test

    Time frame: Baseline through Month 60

  11. Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (SAEs) and Adverse Events of Special Interests (AESIs)

    Time frame: Baseline through Month 60

  12. Change From Baseline Through Month 60 in Creatine Kinase Level

    Time frame: Baseline through Month 60

  13. Time to Change of Loss of Ambulation

    Time frame: Baseline through Month 60

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Registry information

Official study title

A Phase 3 Multinational, Open-label, Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9003 in Subjects With Limb Girdle Muscular Dystrophy 2E/R4

Acronym: EMERGENE

Important dates

Study start
2024
Primary completion
2025
Study completion
2029
First posted
Feb 7, 2024
Registry last updated
Mar 2, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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