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).

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

Sponsors

Lead sponsor: Sarepta Therapeutics, Inc.

Source Sarepta Therapeutics, Inc.
Brief Summary

The proposed clinical trial is the first-in-human, single-center, open label, gene delivery study of SRP-9003 LGMD2E patients. Six total subjects will be enrolled.

Detailed Description

The proposed clinical trial is the first-in-human, single-center, open-label, systemic gene delivery study of SRP-9003 LGMD2E patients. A minimum of six patients will be enrolled. Based on data from Cohort 1, there is a potential to escalate dose for Cohort 2.

Overall Status Active, not recruiting
Start Date October 26, 2018
Completion Date December 31, 2020
Primary Completion Date December 31, 2020
Phase Phase 1/Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Safety based on number of subjects with adverse events (AEs). Baseline upto 3 years
Secondary Outcome
Measure Time Frame
Change From Baseline in Quantity of Beta- Sarcoglycan (SG) Protein Expression at Week 8 Measured by Immunofluorescence Baseline Up to Week 8
Change From Baseline in Quantity of Beta-Sarcoglycan (SG) Protein Expression at Week 8 Measured by Western Blot Baseline Up to Week 8
Enrollment 6
Condition
Intervention

Intervention type: Genetic

Intervention name: SRP-9003

Description: Subjects with diagnosis of LGMD2E will receive SRP-9003 administered through a single systemic injection

Other name: LGMD2E vector

Eligibility

Criteria:

INCLUSION CRITERIA

- Subjects ages 4 through age 15, inclusive

- Males or females of any ethnic group

- Beta-sarcoglycan (SG) DNA gene mutations at both alleles.

- Weakness demonstrated based on history of difficulty in running, jumping and climbing stairs.

- 100 meter walk/run (MWR) test result: >= (greater than equal to) 40 percent (%) of predicted for age, height, gender and weight matched healthy controls at the screening visit.

EXCLUSION CRITERIA

Individuals who meet the following exclusion criteria will not be eligible to participate in the study:

- Active viral infection based on clinical observations.

- Cardiac magnetic resonance imaging (MRI) determined left ventricular ejection fraction less than (<) 40%.

- Serological evidence of human immunodeficiency virus (HIV) infection, or 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 PI creates unnecessary risks for gene transfer.

Other inclusion/exclusion criteria apply.

Gender: All

Minimum age: 4 Years

Maximum age: 15 Years

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Medical Director Study Director Sarepta Therapeutics, Inc.
Location
facility Nationwide Children's Hospital
Location Countries

United States

Verification Date

March 2020

Responsible Party

Responsible party type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Arm group label: Cohort 1

Arm group type: Experimental

Description: Single IV infusion of SRP-9003

Arm group label: Cohort 2

Arm group type: Experimental

Description: Subjects will receive SRP-9003 via intravenous (IV) infusion. Dosage will be determined based on the findings from Cohort 1.

Study Design Info

Allocation: Non-Randomized

Intervention model: Sequential Assignment

Primary purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov