NCT06246513View on ClinicalTrials.gov

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

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

17

Participants

Timeline

Start Date

January 15, 2024

Primary Completion Date

March 4, 2025

Study Completion Date

November 30, 2029

Conditions
Limb-girdle Muscular Dystrophy
Interventions
BIOLOGICAL

SRP-9003

Solution for single IV infusion

DRUG

Glucocorticoid

Oral tablet (prophylactic)

Trial Locations (10)

3000

University Hospital Leuven (UZ Leuven), Leuven

8950

Hospital Sant Joan de Deu, Barcelona

9000

NMRC Gent (UZ Gent), Ghent

19104

The Children's Hospital of Philadelphia, Philadelphia

20122

Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico, Milan

23507

Children's Hospital of The King's Daughter, Norfolk

43205

Nationwide Childrens Hospital, Columbus

45147

Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum, Essen

92037

University of California, San Diego-Altman Clinical and Translational Research Institute, La Jolla

NE1 3BZ

Newcastle University, Newcastle upon Tyne

All Listed Sponsors
lead

Sarepta Therapeutics, Inc.

INDUSTRY

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 | Biotech Hunter | Biotech Hunter