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A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)

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ClinicalTrials.gov Identifier: NCT05876780
Recruitment Status : Active, not recruiting
First Posted : May 25, 2023
Last Update Posted : September 21, 2023
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE May 17, 2023
First Posted Date  ICMJE May 25, 2023
Last Update Posted Date September 21, 2023
Actual Study Start Date  ICMJE December 19, 2022
Estimated Primary Completion Date August 28, 2028   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 17, 2023)
  • Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Month 60 ]
  • Change from Baseline in β-SG Protein Expression Quantity Assessed by Immunofluorescence (IF) Fiber Intensity at Day 60 [ Time Frame: Baseline, Day 60 ]
  • Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Day 60 [ Time Frame: Baseline, Day 60 ]
  • Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Day 60 [ Time Frame: Baseline, Day 60 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 17, 2023)
  • Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Fiber Intensity at Month 24 [ Time Frame: Baseline, Month 24 ]
  • Change from Baseline in β-SG Protein Expression Quantity Assessed by IF Percent Protein Fibers (PPF) at Month 24 [ Time Frame: Baseline, Month 24 ]
  • Change from Baseline in β-SG Protein Expression Quantity Assessed by Western Blot at Month 24 [ Time Frame: Baseline, Month 24 ]
  • Change from Baseline in North Star Assessment for Dysferlinopathy (NSAD) at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Change From Baseline in Time to Rise From the Floor, Time to Complete 100 and 10 meter Walk/Run, and the Timed Stair Ascend 4 Steps Test [ Time Frame: Baseline, Month 60 ]
  • Change from Baseline in Performance of Upper Limb Version 2.0 (PUL 2.0) Score at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Change from Baseline in Ability Captured Through Interactive Video Evaluation (ACTIVE) - Seated Workspace Volume Task at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Cohort 1 (Ambulatory): Change from Baseline in Stride Velocity (95%) and Stair-climbing Velocity (95%) as Assessed by a Wearable Device at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Cohort 1 (Ambulatory): Change from Baseline in Number of Stairs Climbed per Hour as Assessed by a Wearable Device at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Cohort 1 (Ambulatory): Change from Baseline Distance Walked per Hour as Assessed by a Wearable Device at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Cohort 1 (Ambulatory): Change from Baseline in Stride Length (95%) as Assessed by a Wearable Device at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Change from Baseline in Upper Extremity Activity at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Change from Baseline in Angular Wrist Velocity at Month 60 [ Time Frame: Baseline, Month 60 ]
  • Change from Baseline in Vector Genome Copies Using Droplet Digital Polymerase Chain Reaction (ddPCR) in the Target Muscle Tissue [ Time Frame: Baseline, Day 60 and Month 24 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Gene Transfer Single Dose Study to Evaluate the Safety, Tolerability and Efficacy of SRP-9003 in Non-Ambulatory and Ambulatory Participants With Limb Girdle Muscular Dystrophy, Type 2E/R4 (Beta-Sarcoglycan [β-SG] Deficiency)
Official Title  ICMJE A Multicenter, Open-label, Single-dose, Systemic Gene Transfer Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 on Subjects With Limb Girdle Muscular Dystrophy, Type 2E/R4 (β-Sarcoglycan Deficiency)
Brief Summary The primary purpose of this study is to evaluate the safety of SRP-9003 and to quantify expression of β-SG in the skeletal muscle of participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). The study will include both ambulatory (Cohort 1) and non-ambulatory (Cohort 2) participants.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Limb Girdle Muscular Dystrophy
Intervention  ICMJE Genetic: SRP-9003
Single IV infusion of SRP-9003
Other Name: scAAVrh74.MHCK7.hSGCB
Study Arms  ICMJE Experimental: SRP-9003
Participants will receive single IV infusion of SRP-9003 on Day 1.
Intervention: Genetic: SRP-9003
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: September 18, 2023)
6
Original Estimated Enrollment  ICMJE
 (submitted: May 17, 2023)
5
Estimated Study Completion Date  ICMJE August 28, 2028
Estimated Primary Completion Date August 28, 2028   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Cohort 1 only: Ambulatory per protocol specified criteria. - Cohort 2 only: Non-ambulatory per protocol specified criteria and 4 to 50 years of age.
  • Possesses 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-sarcoglycan gene Deoxyribonucleic acid (DNA) (SGCB) gene mutations based on documented clinical findings.
  • Ability to cooperate with muscle testing.

Exclusion Criteria:

  • Presence of any other clinically significant illness or medical condition, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, neuromuscular (other than LGMD2E/R4), or behavioral disease, or infection or malignancy or concomitant illness or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risks for gene transfer or a medical condition or extenuating circumstance that, in the opinion of the Investigator, might compromise the participant's ability to comply with the protocol required testing or procedures or compromise the participant's wellbeing, safety, or clinical interpretability.
  • Exposure to gene therapy, investigational medication, or other protocol-specified treatment within the protocol specified time limits.
  • Any contraindication to use of corticosteroid.

Note: Other inclusion or exclusion criteria could apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 4 Years to 50 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05876780
Other Study ID Numbers  ICMJE SRP-9003-102
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Current Responsible Party Sarepta Therapeutics, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date September 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP