A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)
Sponsored by Sarepta Therapeutics, Inc.
About this trial
Last updated 2 years ago
Study ID
Status
Type
Phase
Placebo
Accepting
Not accepting
Trial Timing
Ended 6 years ago
What is this trial about?
What are the Participation Requirements?
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.