- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03652259
Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency)
February 9, 2024 updated by: Sarepta Therapeutics, Inc.
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)
The proposed clinical trial is the first-in-human, single-center, open-label, gene delivery study of SRP-9003 (bidridistrogene xeboparvovec) in participants with LGMD2E.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
6
Phase
- Phase 2
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Ohio
-
Columbus, Ohio, United States, 43205
- Nationwide Children's Hospital
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
4 years to 15 years (Child)
Accepts Healthy Volunteers
No
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Non-Randomized
- Interventional Model: Sequential Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Cohort 1: SRP-9003
Participants will receive a single intravenous (IV) infusion of SRP-9003 at a prespecified dose.
|
SRP-9003 will be administered through a single systemic injection.
Other Names:
|
Experimental: Cohort 2: SRP-9003
Participants will receive a single IV infusion of SRP-9003.
Dose will be determined based on the findings from Cohort 1.
|
SRP-9003 will be administered through a single systemic injection.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Number of Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
Time Frame: Baseline up to 7 years
|
Baseline up to 7 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in Quantity of Beta-Sarcoglycan (β-SG) Protein Expression at Day 60, as Measured by Western Blot
Time Frame: Baseline, Day 60
|
β-SG gene expression levels will be quantified by Western Blot and compared between pre and post muscle biopsies.
|
Baseline, Day 60
|
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunofluorescence
Time Frame: Baseline, Day 60
|
β-SG gene expression levels will be quantified by immunofluorescence and compared between pre and post muscle biopsies.
|
Baseline, Day 60
|
Change From Baseline in Quantity of β-SG Protein Expression at Day 60, as Measured by Immunohistochemistry Percent B-SG Positive Fibers
Time Frame: Baseline, Day 60
|
Baseline, Day 60
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Medical Director, Sarepta Therapeutics, Inc.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
October 27, 2018
Primary Completion (Estimated)
February 14, 2027
Study Completion (Estimated)
February 14, 2027
Study Registration Dates
First Submitted
August 28, 2018
First Submitted That Met QC Criteria
August 28, 2018
First Posted (Actual)
August 29, 2018
Study Record Updates
Last Update Posted (Estimated)
February 13, 2024
Last Update Submitted That Met QC Criteria
February 9, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Heart Diseases
- Cardiovascular Diseases
- Nervous System Diseases
- Respiratory Tract Diseases
- Respiration Disorders
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Muscular Disorders, Atrophic
- Cardiomyopathies
- Muscular Dystrophies
- Muscular Dystrophies, Limb-Girdle
- Sarcoglycanopathies
Other Study ID Numbers
- SRP-9003-101
- IRB17-00253 (Other Identifier: Sarepta Therapeutics)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Limb-Girdle Muscular Dystrophy, Type 2E
-
Nationwide Children's HospitalMyonexus TherapeuticsRecruitingLimb-Girdle Muscular Dystrophy, Type 2EUnited States
-
Linda Pax LowesRecruitingLimb-Girdle Muscular Dystrophy Type 2A | Limb-Girdle Muscular Dystrophy, Type 2EUnited States
-
Sarepta Therapeutics, Inc.Active, not recruiting
-
University Hospital of North NorwayUniversity of Tromso; Norwegian Muscle Disease Association (FFM); Norwegian National... and other collaboratorsActive, not recruitingMuscular Dystrophies | Limb Girdle Muscular Dystrophy | Limb Girdle Muscular Dystrophy, Type 2I | Limb Girdle Muscular Dystrophy R9 FKRP-relatedNorway
-
ML Bio Solutions, Inc.RecruitingLimb-Girdle Muscular Dystrophy Type 2I (LGMD2I)United States, Netherlands, United Kingdom, Denmark, Australia, Italy, Norway
-
Virginia Commonwealth UniversityUniversity of Colorado, Denver; Washington University School of Medicine; University... and other collaboratorsRecruitingLimb Girdle Muscular Dystrophy | Calpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2A | Limb Girdle Muscular Dystrophy Type R1 | LGMD2AUnited States, United Kingdom
-
Rigshospitalet, DenmarkCompletedBecker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy Type 2IDenmark
-
Cooperative International Neuromuscular Research...Carolinas Medical Center lead study siteCompletedBecker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy, Type 2A (Calpain-3 Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2B (Miyoshi Myopathy, Dysferlin Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2I (FKRP-deficiency)United States
-
IRCCS San Camillo, Venezia, ItalyUniversita di Verona; Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico and other collaboratorsEnrolling by invitationCalpain-3 Deficiency Limb Girdle Muscular Dystrophy Type 2AItaly
-
Mads Peter Godtfeldt StemmerikEdgewise Therapeutics, Inc.CompletedBecker Muscular Dystrophy | McArdle Disease | Limb-Girdle Muscular Dystrophy Type 2Denmark
Clinical Trials on SRP-9003
-
Sarepta Therapeutics, Inc.Active, not recruiting
-
University of BelgradeCompleted
-
University of PisaCompletedDiabetes | PeriodontitisItaly
-
King's College Hospital NHS TrustCompletedPreterm BirthUnited Kingdom, Hong Kong, Chile, Spain, Slovenia, Colombia, Austria, Brazil, Germany, India, Italy, Portugal
-
University of PaviaCompleted
-
University of LjubljanaUniversity Medical Centre LjubljanaRecruitingPeriodontitisSlovenia
-
G. d'Annunzio UniversityNot yet recruitingPeriodontal Diseases | Intrabony Periodontal Defect | Periodontal Attachment Loss | Pocket, Periodontal
-
Sarepta Therapeutics, Inc.RecruitingLimb-girdle Muscular DystrophyUnited States
-
South Rampart Pharma, LLCActive, not recruiting
-
Belén Retamal-ValdesActive, not recruiting