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

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

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Ages Eligible for Study: | 4 Years to 15 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

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
United States, Ohio | |
Nationwide Children's Hospital | |
Columbus, Ohio, United States, 43205 |
Study Director: | Medical Director | Sarepta Therapeutics, Inc. |
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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
limb girdle muscular dystrophy LGMD2E beta-sarcoglycan |
gene transfer adeno-associated virus LGMDR4 |
Muscular Dystrophies Muscular Dystrophies, Limb-Girdle Muscular Disorders, Atrophic Muscular Diseases |
Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn |