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

Phase 1

Completed

• Conditions: Limb-Girdle Muscular Dystrophy, Type 2D
• Interventions: Genetic: SRP-9004

• Registration Number: NCT01976091
• Lead Sponsor: Sarepta Therapeutics, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2013-07-24
• Study First Submitted QC: 2013-10-29
• Study First Posted: 2013-11-05
• Study Start: 2015-02-01
• Primary Completion: 2019-03-14
• Study Completion: 2019-03-14
• Results First Submitted: 2022-03-07
• Results First Submitted QC: 2022-03-07
• Results First Posted: 2022-04-01
• Status Verified: 2023-04
• Last Update Submitted: 2023-05-17
• Last Update Posted: 2023-06-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 6

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1B Low Dose (Bilateral Limb Perfusion)	SRP-9004	Participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to both limbs on Day 0.
Cohort 2 High Dose (Bilateral Limb Perfusion)	SRP-9004	Participants with LGMD2D will receive 1 high dose of SRP-9004 via ILI to both limbs on Day 0.
Cohort 1A: SRP-9004 Low Dose (Single Limb Perfusion)	SRP-9004	Non-ambulant participants with LGMD2D will receive 1 low dose of SRP-9004 via ILI to a single limb on Day 0.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs).	Up to 2 Years	An AE is defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered study drug related. An AE was considered serious if, in the view of the investigator or sponsor, it resulted in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. Treatment-related Treatment Emergent Adverse Event (TEAE) is defined as an TEAE that was classified by the investigator as related to treatment.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline of the Distance Walked in 6 Minutes (6MWT)	Baseline, Up to 2 Years	The 6MWT was performed by standardized procedures for all participants. Participants were asked to walk a set course in 6 minutes (timed), and the distance walked (in meters) was recorded.