Clinical trial NCT04752566: A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Guillain-Barre Syndrome
Keywords: Eculizumab
Soliris
Alexion
C5 Inhibition Therapy
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-02-12
Enrollment: 57
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Time to First Reaching a Hughes Functional Grade (FG) Score <=1
timeFrame: Up to Week 24
Secondary outcomes:
measure: Number of Participants With A Hughes Functional Grade (FG) Score <=1
timeFrame: Week 8, Week 24
measure: Number of Participants With A Hughes Functional Grade Score Improvement of >=3
timeFrame: Week 24
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: Day 1 up to Week 24
measure: Free Complement Component 5 in Serum
timeFrame: Week 24
measure: Hemolytic Complement Activity in Serum
timeFrame: Week 24
measure: Length of Stay in the Hospital
timeFrame: Up to Week 24
measure: Number of Participants Who Required Mechanical Ventilator Support
timeFrame: Up to Week 24
measure: Concentration of Eculizumab in Serum
timeFrame: Up to Week 24
measure: Number of Participants With Positive Antidrug Antibodies
timeFrame: Up to Week 12
Other outcomes:
Locations:
city: Bunkyo-ku
country: Japan
facility: Clinical Trial Site
city: Chiba
country: Japan
facility: Clinical Trial Site
city: Fuchu
country: Japan
facility: Clinical Trial Site
city: Fukuoka
country: Japan
facility: Clinical Trial Site
city: Gifu
country: Japan
facility: Clinical Trial Site
city: Hiroshima
country: Japan
facility: Clinical Trial Site
city: Kagoshima
country: Japan
facility: Clinical Trial Site
city: Kawagoe
country: Japan
facility: Clinical Trial Site
city: Kawasaki
country: Japan
facility: Clinical Trial Site
city: Kitakyushu
country: Japan
facility: Clinical Trial Site
city: Kobe
country: Japan
facility: Clinical Trial Site
city: Kumamoto
country: Japan
facility: Clinical Trial Site
city: Kurashiki
country: Japan
facility: Clinical Trial Site
city: Kyoto
country: Japan
facility: Clinical Trial Site
city: Matsumoto
country: Japan
facility: Clinical Trial Site
city: Mibu
country: Japan
facility: Clinical Trial Site
city: Mitaka
country: Japan
facility: Clinical Trial Site
city: Nagoya
country: Japan
facility: Clinical Trial Site
city: Niigata
country: Japan
facility: Clinical Trial Site
city: Nishinomiya
country: Japan
facility: Clinical Trial Site
city: Osakasayama
country: Japan
facility: Clinical Trial Site
city: Sagamihara
country: Japan
facility: Clinical Trial Site
city: Sapporo
country: Japan
facility: Clinical Trial Site
city: Sendai
country: Japan
facility: Clinical Trial Site
city: Ube
country: Japan
facility: Clinical Trial Site
city: Yokohama
country: Japan
facility: Clinical Trial Site
Study officials:
References: