A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT04752566

Collaborator

(none)

Enrollment

Locations

Arms

19.8

Anticipated Duration (Months)

2.2

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a Phase 3, prospective, multicenter, placebo controlled, double blind, randomized study to investigate the efficacy and safety of eculizumab in participants with severe GBS, defined using the Hughes Functional Grade (FG) scale as progressively deteriorating FG3 or FG4/FG5 within 2 weeks from onset of weakness due to GBS.

This study will be conducted only at sites in Japan.

Condition or Disease	Intervention/Treatment	Phase
Guillain-Barre Syndrome	Biological: Eculizumab Drug: Placebo	Phase 3

Detailed Description

Eligible participants will be randomized to receive intravenous (IV) infusion of eculizumab or placebo at a 2:1 ratio. All participants will be on concomitant IV immunoglobulin G (Ig) therapy as per standard of care.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

57 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Official Title:

A Phase 3, Prospective, Multicenter, Double Blind, Randomized, Placebo Controlled Study to Evaluate the Efficacy and Safety of Eculizumab in Patients With Guillain-Barré Syndrome (GBS)

Actual Study Start Date :

Mar 8, 2021

Anticipated Primary Completion Date :

Oct 31, 2022

Anticipated Study Completion Date :

Oct 31, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Eculizumab Participants will receive eculizumab.	Biological: Eculizumab Eculizumab will be administered via IV infusion once a week for 4 weeks. Other Names: Soliris
Placebo Comparator: Placebo Participants will receive placebo.	Drug: Placebo Placebo will be administered via IV infusion once a week for 4 weeks.

Arm

Intervention/Treatment

Experimental: Eculizumab

Participants will receive eculizumab.

Biological: Eculizumab

Eculizumab will be administered via IV infusion once a week for 4 weeks.

Other Names:

Soliris

Placebo Comparator: Placebo

Participants will receive placebo.

Drug: Placebo

Placebo will be administered via IV infusion once a week for 4 weeks.

Outcome Measures

Primary Outcome Measures

Time To First Reaching A Hughes FG Score ≤ 1 [Up to Week 24]

Secondary Outcome Measures

Proportion Of Participants With A Hughes FG Score ≤ 1 [Week 24]
Proportion Of Participants With A Hughes FG Score Improvement Of ≥ 3 [Week 24]
Proportion Of Participants With A Hughes FG Score ≤ 1 [Week 8]
Incidence Of Treatment-emergent Adverse Events [Up to Week 24]
Free Complement Component 5 In Serum [Week 24]
Hemolytic Complement Activity In Serum [Week 24]
Length Of Stay In The Hospital [Up to Week 24]
Duration Of Ventilator Support [Up to Week 24]
Concentration Of Eculizumab In Serum [Up to Week 24]
Incidence Of Antidrug Antibodies [Up to Week 24]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Participants who meet the GBS criteria.
Participants who were able to run prior to onset of GBS symptoms.
Participants with onset of weakness due to GBS < 2 weeks before screening.
Participants unable to walk unaided for ≥ 5 meters (progressively deteriorating FG3 or FG4 to FG5).
Participants who are already on IVIg or deemed eligible for and who will start IVIg.
Participants who can start their first dose of study drug before the end of the IVIg treatment period.

Exclusion Criteria:

Participants who have previously received or are currently receiving treatment with complement modulators.
Participants who have been administered another investigational product within 30 days or 5 half-lives (whichever is longer) prior to providing consent or are currently participating in another interventional study.
Participants who have received rituximab within 12 weeks prior to screening.
Participants who are being considered for or are already on plasmapheresis.
Participants who have received immunosuppressive treatment during the 4 weeks prior to providing consent.

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Clinical Trial Site	Bunkyo-ku	Japan	113-8519
2	Clinical Trial Site	Chiba	Japan	260-8677
3	Clinical Trial Site	Fuchu	Japan	183-0042
4	Clinical Trial Site	Fukuoka	Japan	814-0180
5	Clinical Trial Site	Gifu	Japan	501-1194
6	Clinical Trial Site	Hiroshima	Japan	730-8518
7	Clinical Trial Site	Kagoshima	Japan	890-8520
8	Clinical Trial Site	Kawagoe	Japan	350-8550
9	Clinical Trial Site	Kawasaki	Japan	216-8511
10	Clinical Trial Site	Kitakyushu	Japan	807-8556
11	Clinical Trial Site	Kobe	Japan	650-0047
12	Clinical Trial Site	Kumamoto	Japan	860-8556
13	Clinical Trial Site	Kurashiki	Japan	710-8602
14	Clinical Trial Site	Kyoto	Japan	602-8566
15	Clinical Trial Site	Matsumoto	Japan	390-8621
16	Clinical Trial Site	Mibu	Japan	321-0293
17	Clinical Trial Site	Mitaka	Japan	181-8611
18	Clinical Trial Site	Nagoya	Japan	466-8560
19	Clinical Trial Site	Niigata	Japan	951-8520
20	Clinical Trial Site	Nishinomiya	Japan	663-8501
21	Clinical Trial Site	Osakasayama	Japan	589-8511
22	Clinical Trial Site	Sagamihara	Japan	252-0375
23	Clinical Trial Site	Sapporo	Japan	060-8648
24	Clinical Trial Site	Sendai	Japan	983-8520
25	Clinical Trial Site	Ube	Japan	755-8505
26	Clinical Trial Site	Yokohama	Japan	236-0004