Trials / Completed
CompletedNCT03375255
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment
- Status
- Completed
- Phase
- Phase 1
- Study type
- Interventional
- Enrollment
- 15 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- Male
- Age
- 12 Years
- Healthy volunteers
- Not accepted
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | SRP-5051 | Single dose of SRP-5051 administered as an intravenous (IV) infusion. |
Timeline
- Start date
- 2018-02-05
- Primary completion
- 2019-08-19
- Completion
- 2019-08-19
- First posted
- 2017-12-18
- Last updated
- 2022-07-06
Locations
8 sites across 2 countries: United States, Canada
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03375255. Inclusion in this directory is not an endorsement.