An Efficacy and Safety Study of Ravulizumab in ALS Participants

Phase 3 Terminated 382 enrolled Alexion Pharmaceuticals, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.

Timeline

Start: 2020-03-30
Primary completion: 2021-10-17
Completion: 2021-10-17

Outcome

Missed primary endpoint

Stopped: “The IDMC recommended the study be discontinued due to lack of efficacy with ravulizumab.”

paper The primary analysis at week 50 showed no significant difference in CAFS between groups (mean [SE], 5.5 [10.8] points; 95% CI, -15.7 to 26.6 points; P = .61). PMID 37695623 ↗

Publications

Genge A, van den Berg LH, Frick G, Han S, Abikoff C, Simmons A, Lin Q, Patra K, Kupperman E, Berry JD. Efficacy and Safety of Ravulizumab, a Complement C5 Inhibitor, in Adults With Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Neurol. 2023 Oct 1;80(10):1089-1097. doi: 10.1001/jamaneurol.2023.2851.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ravulizumab	Monoclonal antibody	—	Intravenous