Phase 3 Study of Dexpramipexole in ALS

Phase 3 Completed 942 enrolled Knopp Biosciences

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of this study is to determine whether dexpramipexole (150 mg twice daily) is safe and effective in the treatment of Amyotrophic Lateral Sclerosis (ALS).

Timeline

Start: 2011-03
Primary completion: 2012-11
Completion: 2012-11

Outcome

Missed primary endpoint

registry analysis (superiority test); Placebo vs Dexpramipexole; p = 0.8568; LS Mean Difference (Final Values) 2.91 (95% CI -28.751 to 34.576); ANCOVA NCT01281189 ↗

paper Dexpramipexole was generally well tolerated but did not differ from placebo on any prespecified efficacy endpoint measurement. PMID 24067398 ↗

release “-- EMPOWER trial fails to demonstrate efficacy in primary and key secondary endpoints --” biogen.com ↗

Publications

Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, Hardiman O, Bozik ME, Ingersoll EW, Archibald D, Meyers AL, Dong Y, Farwell WR, Kerr DA; EMPOWER investigators. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol. 2013 Nov;12(11):1059-67. doi: 10.1016/S1474-4422(13)70221-7. Epub 2013 Sep 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Dexpramipexole	Small molecule	150 mg	Oral