Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
NCT ID: NCT04004065
Last Updated: 2025-03-10
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE2
62 participants
INTERVENTIONAL
2019-06-26
2025-02-07
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Part A: Vesleteplirsen
Participants received escalating dose levels of vesleteplirsen, every 4 weeks, via intravenous (IV) infusion for up to 75 weeks during Part A. Once the doses have been selected for Part B, all participants who have completed Part A will transition to Part B.
Vesleteplirsen
Vesleteplirsen injection, for IV use
Part B: Vesleteplirsen
Participants received vesleteplirsen at the doses selected based on data from Part A every 4 weeks, via IV infusion, for up to 5 years. This included the participants who rolled over from Part A, as well as the additional participants who enrolled at the beginning of Part B.
Vesleteplirsen
Vesleteplirsen injection, for IV use
Interventions
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Vesleteplirsen
Vesleteplirsen injection, for IV use
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Has a genetic diagnosis of Duchenne muscular dystrophy (DMD) and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
* Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight), or has not received corticosteroids for at least 12 weeks prior to study drug administration.
* Has stable pulmonary function (forced vital capacity \[FVC\] ≥40% of predicted and no requirement for nocturnal ventilation).
Exclusion Criteria
* History of hypomagnesemia within 12 weeks prior to Screening.
* Initiation or change of dosing (except for modifications to accommodate changes in weight or changes in standard of care) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, β-blockers, or potassium.
* Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
* Has a left ventricular ejection fraction (LVEF) \<40.0% based on an echocardiogram (ECHO) performed within 12 weeks prior to Screening or at the Screening Visit.
* Treatment with any exon 51-skipping therapy within 4 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.
7 Years
21 Years
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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University of California Davis Health
Sacramento, California, United States
Connecticut Children's
Farmington, Connecticut, United States
Northwest Florida Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
University of Kansas Medical Center Research Inst.
Kansas City, Kansas, United States
University of Massachusetts
Worcester, Massachusetts, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Austin Neuromuscular Center
Austin, Texas, United States
Children's Health Ambulatory Pavilion
Dallas, Texas, United States
Seattle Children's
Seattle, Washington, United States
Universitair Ziekenhuis Gent
Ghent, , Belgium
UZ Leuven
Leuven, , Belgium
Children's Hospital - London Health Sciences Centre (LHSC)
London, Ontario, Canada
University of Essen - Children's Hospital
Essen, , Germany
Klinikum der Universität München
Munich, , Germany
Fondazione Policlinico Universitario A Gemelli
Rome, , Italy
A.O.U. Citta della Salute e della Scienza di Torino - SS Malattie Neuromuscolari, Department of Neurosciences
Torino, , Italy
Leiden University Medical Center
Leiden, , Netherlands
Hospital Sant Joan de Déu. U.B.
Barcelona, , Spain
Hospital Universitari I Politecnic La Fe de Valencia
Valencia, , Spain
Alder Hey Children's NHS Foundation Trust
Liverpool, Lancashire, United Kingdom
Royal Hospital for Children (Glasgow)
Glasgow, , United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, , United Kingdom
Oxford University Hospial NHS Foundation Trust, John Radcliffe Hospital
Oxford, , United Kingdom
Countries
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Other Identifiers
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2019-000601-77
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
5051-201
Identifier Type: -
Identifier Source: org_study_id
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