Overview of eteplirsen (AVI-4658/Exondys51) clinical trials

Eteplirsen (AVI-4658/Exondys51)

Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy

Phase I local treatment (NCT00159250)
• Kinali M, Arechavala-Gomeza V, Feng L, et al. Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. The Lancet. Neurology 2009;8:918-28
Kinali_2009_Lancet Neurol. Oct;8(10);918-28

• Cirak S, Feng L, Anthony K, et al. Restoration of the dystrophin-associated glycoprotein complex after exon skipping therapy in Duchenne muscular dystrophy. Molecular therapy : the journal of the American Society of Gene Therapy 2012;20:462-7.
Cirak_2012_Mol Ther. 2012 Feb;20(2);462-7

 

Phase I/II open label trial (NCT00844597)

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

• Cirak S, Arechavala-Gomeza V, Guglieri M, et al. 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 (London, England) 2011;378:595-605
Cirak_2011_Lancet. Aug 13;378(9791);595-605

 

Phase II placebo-controlled trial (NCT01396239) and 24 wks open-label extension (NCT01540409)

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

Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy

• Mendell JR, Rodino-Klapac LR, Sahenk Z, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Annals of neurology 2013;74:637-47.
Mendell_2013_Ann Neurol. 2013 Nov;74(5);637-47

• Mendell JR, Goemans N, Lowes LP, et al. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Annals of neurology 2016;79:257-71.
Mendell_2016_Ann Neurol. Feb;79(2);257-71

• Kinane TB, Mayer OH, Duda PW, Lowes LP, Moody SL, Mendell JR. Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History. Journal of neuromuscular diseases 2018;5:47-58.
Kinane_2018_J Neuromuscul Dis. ;5(1);47-58

• Charleston JS, Schnell FJ, Dworzak J, et al. Eteplirsen treatment for Duchenne muscular dystrophy: Exon skipping and dystrophin production. Neurology 2018;90:e2146-e2154.
Charleston_2018_Neurology. Jun 12;90(24);e2146-e2154

• Khan N, Eliopoulos H, Han L, et al. Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non Ambulatory Patients with Duchenne Muscular Dystrophy. Journal of neuromuscular diseases 2019

Khan_2019_J Neuromuscul Dis. Mar 7

 

Ongoing trials
• Phase III open-label vs control arm non amenable to exon 51 skipping (NCT02255552; PROMOVI)

Study of Eteplirsen in DMD Patients (PROMOVI)

 

• Phase II open label in early stage (4-6 years) patients (NCT02420379)

Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy

• Phase II open-label in older (7-21 years) patients (NCT02286947)

Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

• Phase II open-label in young (6-48 months) patients (NCT03218995)

Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping

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