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The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 (vesleteplirsen) administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SRP-5051 | Experimental | Patients will be sequentially assigned to receive 1 of the 5 escalating dose levels of SRP-5051 on Day 1. Patients who complete the study and continue to meet safety eligibility criteria will have the opportunity to enroll in an open-label extension study to continue to receive SRP-5051. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SRP-5051 | Drug | Single dose of SRP-5051 administered as an intravenous (IV) infusion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants with Adverse Events (AEs) | An AE is any untoward medical occurrence in a clinical trial participant, which does not necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of the study drug, whether or not considered related to the study drug. | From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks) |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Plasma concentration (Cmax) of SRP-5051 | Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion. | Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours) |
| Area under the plasma concentration versus time curve (AUC) of SRP-5051 |
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Inclusion Criteria:
Exclusion Criteria:
Other inclusion/exclusion criteria apply.
*The dose of steroids must remain constant except for modifications to accommodate changes in weight.
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Sarepta Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neuromuscular Research Center | Sacramento | California | 95817 | United States | ||
| NW FL Clinical Research Group, LLC |
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Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion. |
| Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours) |
| Gulf Breeze |
| Florida |
| 32561 |
| United States |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Children's Medical Center Dallas | Dallas | Texas | 75207 | United States |
| London Health Sciences Centre | London | Ontario | N6A 5W9 | Canada |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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