Phase 2
N=6
A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
Limb-Girdle Muscular Dystrophy, Type 2D
Bottom Line
View on ClinicalTrials.gov: NCT01976091 ↗Enrolled (actual)
6
Serious AEs
50.0%
Results posted
Apr 2022
Primary outcome: Primary: Number of Participants With Adverse Events (AEs). — 1; 3; 2; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- SRP-9004 (Genetic)
- Age
- Pediatric, Adult, Older Adult · 7+ yrs
- Sex
- All
- Sponsor
- Sarepta Therapeutics, Inc.
- Primary completion
- Mar 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events (AEs). |
1; 3; 2; 0; 2; 1 | — |
| SECONDARY Change From Baseline of the Distance Walked in 6 Minutes (6MWT) |
NA; -66.0; 29.0 | — |
Summary
This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D.
Eligibility Criteria
Key Inclusion Criteria
- Cohort 1A must be adult and wheelchair-dependent; Cohorts 1B and 2 will be participants of age 7 or older.
- Confirmed alpha-sarcoglycan deficiency or identified sarcoglycan alpha (SGCA) deoxyribonucleic acid (DNA) mutation.
- Participants enrolled in Cohorts 1B or 2 must be able to walk independently, but must exhibit signs of lower extremity weakness (that is, a Gowers' sign, use a handrail for climbing stairs) and walk ≤80% of predicted distance on the 6 minute walk test (6MWT) based on normative data.
Key Exclusion Criteria
- Active viral infection based on clinical observations.
- The presence of SGCA mutations without weakness or loss of function.
- Symptoms or signs of cardiomyopathy.
- Serological evidence of human immunodeficiency virus (HIV), Hepatitis B, or C infection.
- Diagnosis of (or ongoing treatment for) an autoimmune disease.
- Participants with AAVrh74 or AAV8 binding antibody titers ≥ 1:50 as determined by enzyme-linked immunosorbent assay (ELISA) immunoassay.
Other inclusion/exclusion criteria apply.
Data sourced from ClinicalTrials.gov (NCT01976091). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.