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

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ClinicalTrials.gov Identifier: NCT03652259
Recruitment Status : Active, not recruiting
First Posted : August 29, 2018
Last Update Posted : October 26, 2020
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 LGMD2E patients. Up to Six patients will be enrolled.

Condition or disease Intervention/treatment Phase
Limb-Girdle Muscular Dystrophy, Type 2E Genetic: SRP-9003 Phase 1 Phase 2

Detailed Description:
The proposed clinical trial is the first-in-human, single-center, open-label, systemic gene delivery study of SRP-9003 LGMD2E patients. Up to six patients will be enrolled in 2 cohorts. Based on data from Cohort 1, there is a potential to escalate dose for Cohort 2. LGMD2E is also referred to as LGMDR4.
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 2023
Estimated Study Completion Date : February 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Cohort 1
Single IV infusion of SRP-9003.
 Genetic: SRP-9003
Patients with diagnosis of LGMD2E will receive SRP-9003 administered through a single systemic injection.
Other Name: LGMD2E vector
Experimental: Cohort 2
Patients will receive SRP-9003 via intravenous (IV) infusion. Dosage will be determined based on the findings from Cohort 1.
 Genetic: SRP-9003
Patients with diagnosis of LGMD2E will receive SRP-9003 administered through a single systemic injection.
Other Name: LGMD2E vector


Outcome Measures
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Primary Outcome Measures :
  1. Safety Based on Number of Patients With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (SAEs) [ Time Frame: Baseline to 3 years ]

Secondary Outcome Measures :
  1. Change From Baseline in Quantity of Beta-Sarcoglycan (B-SG) Protein Expression Measured by Western Blot [ Time Frame: Baseline to Week 8 ]
    Beta-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 Beta- Sarcoglycan (B-SG) Protein Expression Measured by Immunofluorescence [ Time Frame: Baseline to Week 8 ]
    Beta-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.

  3. Change From Baseline in Quantity of Beta- Sarcoglycan (B-SG) Protein Expression Measured by Immunohistochemistry Percent B-SG Positive Fibers [ Time Frame: Baseline to Week 8 ]

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

  • Patients ages 4 through age 15, inclusive.
  • Males or females of any ethnic group.
  • Beta-sarcoglycan (B-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.

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: October 26, 2020
Last Verified: October 2020

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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
Muscular Disorders, Atrophic
Muscular Diseases
 Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn