%0 Journal Article %@ 1929-0748 %I JMIR Publications %V 5 %N 4 %P e210 %T A Prospective, Multicenter, Randomized Phase II Study to Evaluate the Efficacy and Safety of Eculizumab in Patients with Guillain-Barré Syndrome (GBS): Protocol of Japanese Eculizumab Trial for GBS (JET-GBS) %A Yamaguchi,Nobuko %A Misawa,Sonoko %A Sato,Yasunori %A Nagashima,Kengo %A Katayama,Kanako %A Sekiguchi,Yukari %A Iwai,Yuta %A Amino,Hiroshi %A Suichi,Tomoki %A Yokota,Takanori %A Nishida,Yoichiro %A Kohara,Nobuo %A Hirata,Koichi %A Nishiyama,Kazutoshi %A Yabe,Ichiro %A Kaida,Ken-Ichi %A Suzuki,Norihiro %A Nodera,Hiroyuki %A Tsuji,Shoji %A Koike,Haruki %A Kira,Jun-Ichi %A Hanaoka,Hideki %A Kusunoki,Susumu %A Kuwabara,Satoshi %A , %+ Department of Neurology, Chiba University Graduate School of Medicine, Inohana 1-8-1, Chuo-ku, Chiba, Japan, 81 43 222 7171 ext 5414, sonoko.m@mb.infoweb.ne.jp %K Guillain-Barré syndrome %K eculizumab %K complement activation %K clinical trial %K antiganglioside antibody %D 2016 %7 07.11.2016 %9 Protocol %J JMIR Res Protoc %G English %X Background: Guillain-Barré syndrome (GBS) is an immune-mediated neuropathy that causes acute flaccid paralysis. Immunoglobulin and plasma exchange are established treatments for GBS; however, a substantial number of patients, particularly those with severe disease, have poor recovery and residual deficits. Recent studies suggest that complement activation plays a pivotal role in GBS-associated axonal degeneration, and eculizumab is a humanized monoclonal antibody that specifically binds to complement component 5 and potently inhibits complement activation. Objective: This clinical trial aims to evaluate the efficacy and safety of eculizumab, a humanized monoclonal antibody directed against complement component 5, for treatment of GBS. Methods: The Japanese Eculizumab Trial for GBS (JET-GBS) is a prospective, multicenter, placebo-controlled, double-blind, randomized phase II study conducted at 13 tertiary neurology centers and is funded by the Japan Agency for Medical Research and Development. A total of 33 GBS patients unable to walk independently within 2 weeks from symptom onset (Hughes functional grade 3-5) were randomized at a 2:1 ratio to receive either intravenous eculizumab (900 mg/day) or placebo once weekly for 4 weeks, followed by 20 weeks of follow-up. The primary endpoint for efficacy is the proportion of patients who regain their ability to walk without aid at 4 weeks after the first dose of the study treatment, while primary safety outcomes are the incidence of adverse events and serious adverse events during the trial. Results: Enrollment for the trial began in August 2015. This trial is still ongoing. All participants have been enrolled, and follow-up will be completed in October 2016. Conclusions: This study is the first to investigate the efficacy and safety of eculizumab for GBS. In case of a positive result, we will plan a phase III trial to investigate this issue in a larger number of patients. ClinicalTrial: UMIN Clinical Trials Registry UMIN 000018171; https:/upload.umin.ac.jp/cgi-open-bin/ctr/ctr.cgi?function= brows&action=brows&type=summary&language=J&recptno=R000020978 (Archived by WebCite at http://www.webcitation.org/ 6lTiG8ltG). Clinical Trials.gov NCT02493725; https://clinicaltrials.gov/ct2/show/NCT02493725 (Archived by WebCite at http://www.webcitation.org/6lVJZXKSL) %M 27821382 %R 10.2196/resprot.6610 %U http://www.researchprotocols.org/2016/4/e210/ %U https://doi.org/10.2196/resprot.6610 %U http://www.ncbi.nlm.nih.gov/pubmed/27821382