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Gene Delivery Clinical Trial of SRP-9003 for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03652259
Recruitment Status : Active, not recruiting
First Posted : August 29, 2018
Last Update Posted : June 8, 2021
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Study Description
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Brief Summary:
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 in participants with LGMD2E.

Condition or disease Intervention/treatment Phase
Limb-Girdle Muscular Dystrophy, Type 2E Genetic: SRP-9003 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: A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency)
Actual Study Start Date : October 27, 2018
Estimated Primary Completion Date : February 14, 2025
Estimated Study Completion Date : February 14, 2025

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1: SRP-9003
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.
 Genetic: SRP-9003
SRP-9003 will be administered through a single systemic injection.
Other Name: LGMD2E vector
Experimental: Cohort 2: SRP-9003
Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
 Genetic: SRP-9003
SRP-9003 will be administered through a single systemic injection.
Other Name: LGMD2E vector


Outcome Measures
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Primary Outcome Measures :
  1. Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) [ Time Frame: Baseline up to 5 years ]

Secondary Outcome Measures :
  1. Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot [ Time Frame: Baseline, Day 60 ]
    β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.

  2. Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence [ Time Frame: Baseline, Day 60 ]
    β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.

  3. Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers [ Time Frame: Baseline, Day 60 ]

Eligibility Criteria
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Ages Eligible for Study:   4 Years to 15 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

INCLUSION CRITERIA

  • Males or females of any ethnic group
  • β-SG deoxyribonucleic acid (DNA) gene mutations at both alleles
  • Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs
  • A 100 meter walk/run (MWR) test result: ≥40 % of predicted for age-, height-, gender-, and weight-matched healthy controls at the screening visit

EXCLUSION CRITERIA

  • Active viral infection based on clinical observations
  • Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction (LVEF) <40%
  • Serological evidence of human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection
  • Diagnosis of (or ongoing treatment for) an autoimmune disease
  • Abnormal laboratory values considered clinically significant
  • Concomitant illness or requirement for chronic drug treatment that, in the opinion of the Principal Investigator, creates unnecessary risks for gene transfer.

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): NCT03652259


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
More Information
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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03652259    
Other Study ID Numbers: SRP-9003-101
IRB17-00253 ( Other Identifier: Sarepta Therapeutics )
First Posted: August 29, 2018    Key Record Dates
Last Update Posted: June 8, 2021
Last Verified: June 2021

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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
LGMD2E
beta-sarcoglycan
 gene transfer
adeno-associated virus
LGMDR4
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