Trials / Completed
CompletedNCT03218995
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 15 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- Male
- Age
- 6 Months – 48 Months
- Healthy volunteers
- Not accepted
Summary
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Eteplirsen | Infusion for intravenous use. |
Timeline
- Start date
- 2017-08-16
- Primary completion
- 2021-03-10
- Completion
- 2021-03-10
- First posted
- 2017-07-17
- Last updated
- 2021-12-09
- Results posted
- 2021-12-09
Locations
4 sites across 4 countries: Belgium, France, Italy, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT03218995. Inclusion in this directory is not an endorsement.