Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

Phase 1/2 Completed 19 enrolled Sarepta Therapeutics, Inc. British Medical Research Council · collab

Non-randomizedSingle-groupOpen-labelTreatment

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).

Timeline

Start: 2009-01
Primary completion: 2010-06
Completion: 2010-12

Publications

Results Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, Dickson G, Wood MJ, Wilton SD, Straub V, Kole R, Shrewsbury SB, Sewry C, Morgan JE, Bushby K, Muntoni F. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet. 2011 Aug 13;378(9791):595-605. doi: 10.1016/S0140-6736(11)60756-3. Epub 2011 Jul 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Eteplirsen	Antisense oligonucleotide	0.5 mg/kg	Intravenous
Subject	Eteplirsen	Antisense oligonucleotide	1 mg/kg	Intravenous
Subject	Eteplirsen	Antisense oligonucleotide	2 mg/kg	Intravenous
Subject	Eteplirsen	Antisense oligonucleotide	4 mg/kg	Intravenous
Subject	Eteplirsen	Antisense oligonucleotide	10 mg/kg	Intravenous
Subject	Eteplirsen	Antisense oligonucleotide	20 mg/kg	Intravenous

Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients

Summary

Timeline

Publications

Drugs

Indications