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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-02-01

Study Completion Date

2019-03-14

Brief Summary

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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.

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Detailed Description

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Conditions

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Limb-Girdle Muscular Dystrophy, Type 2D

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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

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

Group Type EXPERIMENTAL

SRP-9004

Intervention Type GENETIC

Isolated Limb Infusion (ILI)

Cohort 1B Low Dose (Bilateral Limb Perfusion)

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

Group Type EXPERIMENTAL

SRP-9004

Intervention Type GENETIC

Isolated Limb Infusion (ILI)

Cohort 2 High Dose (Bilateral Limb Perfusion)

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

Group Type EXPERIMENTAL

SRP-9004

Intervention Type GENETIC

Isolated Limb Infusion (ILI)

Interventions

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SRP-9004

Isolated Limb Infusion (ILI)

Intervention Type GENETIC

Other Intervention Names

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patidistrogene bexoparvovec

Eligibility Criteria

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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.

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.

Minimum Eligible Age

7 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No