TY  - JOUR
AU  - Yamaguchi, Nobuko
AU  - Misawa, Sonoko
AU  - Sato, Yasunori
AU  - Nagashima, Kengo
AU  - Katayama, Kanako
AU  - Sekiguchi, Yukari
AU  - Iwai, Yuta
AU  - Amino, Hiroshi
AU  - Suichi, Tomoki
AU  - Yokota, Takanori
AU  - Nishida, Yoichiro
AU  - Kohara, Nobuo
AU  - Hirata, Koichi
AU  - Nishiyama, Kazutoshi
AU  - Yabe, Ichiro
AU  - Kaida, Ken-Ichi
AU  - Suzuki, Norihiro
AU  - Nodera, Hiroyuki
AU  - Tsuji, Shoji
AU  - Koike, Haruki
AU  - Kira, Jun-Ichi
AU  - Hanaoka, Hideki
AU  - Kusunoki, Susumu
AU  - Kuwabara, Satoshi
PY  - 2016
DA  - 2016/11/07
TI  - 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)
JO  - JMIR Res Protoc
SP  - e210
VL  - 5
IS  - 4
KW  - Guillain-Barré syndrome
KW  - eculizumab
KW  - complement activation
KW  - clinical trial
KW  - antiganglioside antibody
AB  - 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) 
SN  - 1929-0748
UR  - http://www.researchprotocols.org/2016/4/e210/
UR  - https://doi.org/10.2196/resprot.6610
UR  - http://www.ncbi.nlm.nih.gov/pubmed/27821382
DO  - 10.2196/resprot.6610
ID  - info:doi/10.2196/resprot.6610
ER  -