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

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ClinicalTrials.gov Identifier: NCT01976091
Recruitment Status : Completed
First Posted : November 5, 2013
Results First Posted : April 1, 2022
Last Update Posted : September 13, 2022
Sponsor:
Collaborator:
Nationwide Children's Hospital
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Limb-Girdle Muscular Dystrophy, Type 2D Genetic: SRP-9004 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase I/IIA Gene Transfer Clinical Trial for LGMD2D (Alpha-Sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA
Actual Study Start Date : February 1, 2015
Actual Primary Completion Date : March 14, 2019
Actual Study Completion Date : March 14, 2019

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: 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.
 Genetic: SRP-9004
Isolated Limb Infusion (ILI)
Other Name: patidistrogene bexoparvovec
Experimental: 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.
 Genetic: SRP-9004
Isolated Limb Infusion (ILI)
Other Name: patidistrogene bexoparvovec
Experimental: 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.
 Genetic: SRP-9004
Isolated Limb Infusion (ILI)
Other Name: patidistrogene bexoparvovec


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs). [ Time Frame: 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 :
  1. Change From Baseline of the Distance Walked in 6 Minutes (6MWT) [ Time Frame: 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.


Eligibility Criteria
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Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01976091


Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Nationwide Children's Hospital
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
  Study Documents (Full-Text)

Documents provided by Sarepta Therapeutics, Inc.:
Study Protocol  [PDF] February 10, 2017
Statistical Analysis Plan  [PDF] April 25, 2022

More Information
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Publications:

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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT01976091    
Other Study ID Numbers: 9004-101
5U01AR060911 ( U.S. NIH Grant/Contract )
First Posted: November 5, 2013    Key Record Dates
Results First Posted: April 1, 2022
Last Update Posted: September 13, 2022
Last Verified: August 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sarepta Therapeutics, Inc.:
limb girdle muscular dystrophy
LGMD2D
alpha-sarcoglycan
gene transfer
adeno-associated virus
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophies, Limb-Girdle
Sarcoglycanopathies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
 Nervous System Diseases
Genetic Diseases, Inborn
Respiration Disorders
Respiratory Tract Diseases
Cardiomyopathies
Heart Diseases
Cardiovascular Diseases