One Time Gene Therapy Infusion for Patients With LGMD2D/R3
Phase: Recruiting
First Posted: March
Condition(s): Limb Girdle Muscular Dystrophy 2D/R3
Other Study ID Number(s): SRP-9004-102
What Is the Purpose of This Study?
Assess the safety of SRP-9004
Who Can Take Part in This Study?
- Patients that are 4 years and older
- ≤70 kilograms
- Possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic α-SG deoxyribonucleic acid (DNA) gene mutations as documented prior to screening
- Participants must have adeno-associated virus (AAV) serotype Rh74 (rh74) antibody titers <1:400 (that is, not elevated) as determined by an enzyme-linked immunosorbent assay (ELISA).
- Required functional testing scores
For a full list of eligibility requirements, visit https://clinicaltrials.gov/study/NCT06747273
What Will Happen During This Study?
- Participants will receive a single dose of SRP-9004 via intravenous (IV) infusion
- Each participant's study duration is 5 years
- Muscle biopsy
- Strength and functional testing
Principal Investigator
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