Trials / Completed
CompletedNCT02420379
Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 33 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- Male
- Age
- 4 Years – 6 Years
- Healthy volunteers
- Not accepted
Summary
This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Detailed description
Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients. Clinical efficacy, including functional tests and MRI, will be assessed at regularly scheduled study visits. Patients will undergo one baseline and one follow-up muscle biopsy. Population and serial PK will be collected.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | eteplirsen | Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks. |
Timeline
- Start date
- 2015-06-30
- Primary completion
- 2018-12-17
- Completion
- 2018-12-17
- First posted
- 2015-04-17
- Last updated
- 2021-01-25
- Results posted
- 2020-07-22
Locations
13 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT02420379. Inclusion in this directory is not an endorsement.