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 (EMERGENE)

Limb-girdle Muscular Dystrophy Clinical Trial

Official 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06246513
• Other study ID #: SRP-9003-301
• Secondary ID: 2022-503112-17-0
• Status: Recruiting
• Phase: Phase 3
• Start date: January 15, 2024
• Est. completion date: November 30, 2029

Study information

• Verified date: May 2024
• Source: Sarepta Therapeutics, Inc.
• Contact: Sarepta Therapeutics Inc. For Clinical Trial Information, Select
• Phone: 1-888-SAREPTA (1-888-727-3782)
• Email: SareptAlly[@]sarepta.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: November 30, 2029
• Est. primary completion date: January 31, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

Eligibility

Inclusion Criteria:

• Cohort 1, only ambulatory participants:

• Able to walk without assistive aid

• 10-meter walk test (10MWT) <30 seconds

• NSAD =25

• Cohort 2, only non-ambulatory participants:

• 10MWT =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 ß-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.

Related Conditions & MeSH terms

• Limb-girdle Muscular Dystrophy
• Muscular Dystrophies

Intervention

Biological:
• SRP-9003
Solution for single IV infusion

Drug:
• Glucocorticoid
Oral tablet (prophylactic)

Locations

Country	Name	City	State
United States	Nationwide Childrens Hospital	Columbus	Ohio
United States	University of California, San Diego-Altman Clinical and Translational Research Institute	La Jolla	California
United States	Children's Hospital of The King's Daughter	Norfolk	Virginia
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Sarepta Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in ß-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent ß-SG Positive Fibers		Baseline, Day 60
Secondary	Change From Baseline in ß-SG Expression at Day 60 Post-dose as Measured by Western Blot		Baseline, Day 60
Secondary	Change From Baseline Through Month 60 in North Star Assessment for Dysferlinopathy (NSAD) Total Score		Baseline through Month 60
Secondary	Change From Baseline Through Month 60 in Performance of Upper Limb Version 2.0 (PUL 2.0) Total Score		Baseline through Month 60
Secondary	Cohort 1: Change From Baseline Through Month 60 in the Time to Rise from the Floor Test		Baseline through Month 60
Secondary	Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 10-meter Walk/Run (10MWR) Test		Baseline through Month 60
Secondary	Cohort 1: Change From Baseline Through Month 60 in the Time to Ascend 4 Steps Test		Baseline through Month 60
Secondary	Cohort 1: Change From Baseline Through Month 60 in the Time to Complete the 100-meter Walk/Run (100MWR) Test		Baseline through Month 60
Secondary	Cohort 1: Change From Baseline Through Month 60 in the Timed Up and Go Test		Baseline through Month 60
Secondary	Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs)		Baseline through Month 60
Secondary	Change From Baseline Through Month 60 in Creatine Kinase Level		Baseline through Month 60
Secondary	Time to Change of Loss of Ambulation		Baseline through Month 60
Secondary	Change from Baseline in ß-SG Expression at Day 60 Post-dose as Measured by IF Percent Fluorescent Expression		Baseline, Day 60