Back to resultsGene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
Primary Purpose
Limb-Girdle Muscular Dystrophy, Type 2E
Status
Active
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
SRP-9003
Sponsored by
About this trial
This is an interventional treatment trial for Limb-Girdle Muscular Dystrophy, Type 2E focused on measuring limb girdle muscular dystrophy, LGMD2E, beta-sarcoglycan, gene transfer, adeno-associated virus, LGMDR4
Eligibility 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.
Sites / Locations
- Nationwide Children's Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Cohort 1: SRP-9003
Cohort 2: SRP-9003
Arm Description
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.
Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
Outcomes
Primary Outcome Measures
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Secondary Outcome Measures
Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot
β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence
β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers
Full Information
NCT ID
NCT03652259
First Posted
August 28, 2018
Last Updated
September 28, 2022
Sponsor
Sarepta Therapeutics, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03652259
Brief Title
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
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)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 27, 2018 (Actual)
Primary Completion Date
February 14, 2025 (Anticipated)
Study Completion Date
February 14, 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sarepta Therapeutics, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Limb-Girdle Muscular Dystrophy, Type 2E
Keywords
limb girdle muscular dystrophy, LGMD2E, beta-sarcoglycan, gene transfer, adeno-associated virus, LGMDR4
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort 1: SRP-9003
Arm Type
Experimental
Arm Description
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.
Arm Title
Cohort 2: SRP-9003
Arm Type
Experimental
Arm Description
Participants will receive a single IV infusion of SRP-9003. Dose will be determined based on the findings from Cohort 1.
Intervention Type
Genetic
Intervention Name(s)
SRP-9003
Other Intervention Name(s)
LGMD2E vector, bidridistrogene xeboparvovec
Intervention Description
SRP-9003 will be administered through a single systemic injection.
Primary Outcome Measure Information:
Title
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Time Frame
Baseline up to 5 years
Secondary Outcome Measure Information:
Title
Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot
Description
β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.
Time Frame
Baseline, Day 60
Title
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence
Description
β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.
Time Frame
Baseline, Day 60
Title
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
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
15 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Sarepta Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
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