NCT01396239

Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients

A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658(Eteplirsen),in the Treatment of Ambulant Subjects With Duchenne Muscular Dystrophy

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 7 Years – 13 Years
• Healthy volunteers: Not accepted

Summary

This study is designed to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of AVI-4658 (eteplirsen) in both 50.0 mg/kg and 30.0 mg/kg doses administered over 24 weeks in subjects diagnosed with Duchenne muscular dystrophy (DMD).

Detailed description

For details regarding the roll-over extension study 4658-us-202, please refer to NCT01540409.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
DRUG	AVI-4658 (Eteplirsen)	Treatment group 1 (n=4): 50.0 mg/kg eteplirsen once weekly x 24 weeks via a 60-minute i.v. infusion Treatment group 2 (n=4): 30.0 mg/kg eteplirsen once weekly x 24 weeks via a 60-minute i.v. infusion Treatment group 3 (n=4): matching placebo once weekly x 24 weeks via a 60-minute i.v. infusion; treatment group 3a will match two placebo subjects to 50.0 mg/kg eteplirsen; treatment group 3b will match two placebo subjects to 30.0 mg/kg eteplirsen
OTHER	Placebo	sterile, isotonic, clear, colorless phosphate buffered saline solution of eteplirsen at a concentration of 100 mg/mL in single-use vials containing a nominal volume of 1.0 mL without preservatives.

Timeline

• Start date: 2011-07-01
• Primary completion: 2012-02-01
• Completion: 2012-06-01
• First posted: 2011-07-18
• Last updated: 2020-03-30
• Results posted: 2015-11-09

Locations

1 site across 1 country: United States