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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 : Recruiting
First Posted : August 29, 2018
Last Update Posted : October 25, 2019
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
Limb-Girdle Muscular Dystrophy, Type 2E Drug: 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. A minimum of six patients will be enrolled. Based on data from Cohort 1, there is a potential to escalate dose for Cohort 2.

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Study Type : Interventional  (Clinical Trial)
Estimated 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 26, 2018
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : December 31, 2020


Arm Intervention/treatment
Experimental: Cohort 1
Single IV infusion of SRP-9003
Drug: SRP-9003
Subjects with diagnosis of LGMD2E will receive SRP-9003 administered through a single systemic injection
Other Name: LGMD2E vector

Experimental: Cohort 2
Subjects will receive SRP-9003 via intravenous (IV) infusion. Dosage will be determined based on the findings from Cohort 1.
Drug: SRP-9003
Subjects with diagnosis of LGMD2E will receive SRP-9003 administered through a single systemic injection
Other Name: LGMD2E vector




Primary Outcome Measures :
  1. Safety based on number of subjects with adverse events (AEs). [ Time Frame: Baseline upto 3 years ]
    AEs will be monitored and scored for severity and relatedness to the study article.


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

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



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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

  • 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.


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


Contacts
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Contact: Medical Information 1-888-727-3782 clinicaltrials@sarepta.com

Locations
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United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Irene Panagopoulos, MS    614-355-2602    Irene.Panagopoulos@nationwidechildrens.org   
Principal Investigator: Jerry R Mendell, M.D.         
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.

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Responsible Party: Sarepta Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03652259     History of Changes
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 25, 2019
Last Verified: October 2019

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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
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