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NCT03375255 · ClinicalTrials.gov registry record · Phase 1
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 study of Muscular Dystrophy, Duchenne, sponsored by Sarepta Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 15
- Enrollment target
- 8
- Study locations
NCT03375255: Completed Phase 1 study of Muscular Dystrophy, Duchenne, sponsored by Sarepta Therapeutics.
NCT03375255 is a Phase 1 study of Muscular Dystrophy, Duchenne that has completed, run by Sarepta Therapeutics. The registered enrollment target is 15 participants, below the 104-participant average among 4 other Muscular Dystrophy, Duchenne trials with a reported enrollment target (86% lower). The trial reports 8 study locations across 8 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03375255, a Phase 1 study of Muscular Dystrophy, Duchenne, has completed, sponsored by Sarepta Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 15 participants
- Enrollment target
- 8
- Study locations
Study 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.
Primary Outcome
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.
Conditions Studied
Interventions
- DRUG SRP-5051
Study Locations (8)
California
- Neuromuscular Research Center - Sacramento
Florida
- NW FL Clinical Research Group, LLC - Gulf Breeze
Georgia
- Rare Disease Research, LLC - Atlanta
Illinois
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Kansas
- University of Kansas Medical Center - Kansas City
Pennsylvania
- Children's Hospital of Pittsburgh of UPMC - Pittsburgh
Texas
- Children's Medical Center Dallas - Dallas
Ontario
- London Health Sciences Centre - London
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 15 participants |
| Start Date | 2018-02-05 |
| Est. Completion | 2019-08-19 |
| Phase | Phase 1 |
What the finished NCT03375255 record still lists
NCT03375255 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 15 participants, a relatively small participant target, below the 104-participant average among 4 other Muscular Dystrophy, Duchenne trials with a reported enrollment target (86% lower).
The record links to 1 condition, with Muscular Dystrophy, Duchenne appearing as the primary indexed condition, and to 1 intervention - of which SRP-5051 is the first listed.
NCT03375255 lists 8 locations in 8 states (California, Florida, Georgia).
Frequently Asked Questions
What is clinical trial NCT03375255 about?
NCT03375255 is a clinical study titled "A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)". 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.
What is the current status of trial NCT03375255?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2018-02-05. Estimated completion is 2019-08-19.
What conditions does trial NCT03375255 study?
This clinical trial studies the following conditions: Muscular Dystrophy, Duchenne.
What interventions are being tested in trial NCT03375255?
The interventions under investigation include: SRP-5051 (DRUG).
Who is sponsoring clinical trial NCT03375255?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03375255 being conducted?
This trial has 8 study locations across California, Florida, Georgia, Illinois, Kansas. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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