Gene Delivery Clinical Trial of SRP-9003 for Patients With LGMD2E (Beta-sarcoglycan Deficiency)

Brief Title

Gene Delivery Clinical Trial of SRP-9003 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)

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.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)

Secondary Outcome

 Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot

Condition

Limb-Girdle Muscular Dystrophy, Type 2E

Intervention

SRP-9003

Study Arms / Comparison Groups

 Cohort 1: SRP-9003
Description:  Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Genetic

Estimated Enrollment

6

Start Date

October 27, 2018

Completion Date

February 14, 2025

Primary Completion Date

February 14, 2025

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.
      

Gender

All

Ages

4 Years - 15 Years

Accepts Healthy Volunteers

No

Contacts

Medical Director, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03652259

Organization ID

SRP-9003-101

Secondary IDs

IRB17-00253

Responsible Party

Sponsor

Study Sponsor

Sarepta Therapeutics, Inc.


Study Sponsor

Medical Director, Study Director, Sarepta Therapeutics, Inc.


Verification Date

June 2021