Trials / Completed
CompletedNCT00844597
Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
Clinical Study to Assess the Safety fo AVI-4658 in Subjects With Duchenne Muscular Dystrophy Due to a Frame-shift Mutation Amenable to Correction by Skipping Exon 51.
- Status
- Completed
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 19 (actual)
- Sponsor
- Sarepta Therapeutics, Inc. · Industry
- Sex
- Male
- Age
- 5 Years – 15 Years
- Healthy volunteers
- Not accepted
Summary
The specific aim of this Phase I/II study is to assess the safety of intravenous administered Morpholino oligomer directed against exon 51 (AVI-4658 PMO).
Detailed description
Primary outcome is safety, tolerability and dose selection for future studies.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | AVI-4658 for Injection | AVI-4658 for Injection, is packaged as 100 mg/mL in phosphate buffered saline with 1 mL per vial. Study dosages will be infused over a 1 hour period with Normal saline in 6 dose cohorts. |
Timeline
- Start date
- 2009-01-01
- Primary completion
- 2010-06-01
- Completion
- 2010-12-01
- First posted
- 2009-02-16
- Last updated
- 2015-10-06
- Results posted
- 2015-10-06
Locations
2 sites across 1 country: United Kingdom
Source: ClinicalTrials.gov record NCT00844597. Inclusion in this directory is not an endorsement.