A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 (Patidistrogene Bexoparvovec) in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D)

Sarepta Therapeutics

Status and phase: Completed; Phase 2; Phase 1

Conditions: Limb-Girdle Muscular Dystrophy, Type 2D

Treatments: Genetic: SRP-9004

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01976091

9004-101

5U01AR060911

Details and patient eligibility

About

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.

Enrollment: 6 patients

Sex: All

Ages: 7+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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6 participants in 3 patient groups

Cohort 1A: SRP-9004 Low Dose (Single Limb Perfusion)

Experimental group

Description:

Non-ambulant participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to a single limb on Day 0.

Treatment:

Genetic: SRP-9004

Cohort 1B Low Dose (Bilateral Limb Perfusion)

Experimental group

Description:

Participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to both limbs on Day 0.

Treatment:

Genetic: SRP-9004

Cohort 2 High Dose (Bilateral Limb Perfusion)

Experimental group

Description:

Participants with LGMD2D will receive 1 high dose of SRP-9004 via ILI to both limbs on Day 0.

Treatment:

Genetic: SRP-9004