A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy

Phase 3 Completed 186 enrolled GlaxoSmithKline

RandomizedSingle-groupQuadruple-blindTreatment

Summary

The purpose of this study is to determine whether GSK2402968 is effective in the treatment of ambulant boys with Duchenne muscular dystrophy resulting from a mutation thought to be corrected by exon 51 skipping.

Timeline

Start: 2010-12-02
Primary completion: 2013-06-28
Completion: 2013-06-28

Outcome

Missed primary endpoint

registry analysis (superiority test); Placebo vs GSK2402968 6mg/kg/Week; p = 0.415; Mean Difference (Net) 10.334 (95% CI -14.645 to 35.312) NCT01254019 ↗

paper A nonsignificant treatment difference (P = 0.415) in the change from baseline in six-minute walk distance (6MWD; primary efficacy endpoint) PMID 29203355 ↗

release “drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy (DMD) patients with an amenable mutation, did not meet the primary endpoint” gsk.com ↗

Publications

Goemans N, Mercuri E, Belousova E, Komaki H, Dubrovsky A, McDonald CM, Kraus JE, Lourbakos A, Lin Z, Campion G, Wang SX, Campbell C; DEMAND III study group. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy. Neuromuscul Disord. 2018 Jan;28(1):4-15. doi: 10.1016/j.nmd.2017.10.004. Epub 2017 Dec 6.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Drisapersen	Antisense oligonucleotide	6 mg/kg	Subcutaneous