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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04752566
Recruitment Status : Active, not recruiting
First Posted : February 12, 2021
Last Update Posted : April 12, 2022
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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.

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Study Type : Interventional  (Clinical Trial)
Estimated 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 : March 8, 2021
Estimated Primary Completion Date : October 31, 2022
Estimated Study Completion Date : October 31, 2022

Resource links provided by the National Library of Medicine

Drug Information available for: Eculizumab

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 Name: Soliris

Placebo Comparator: Placebo
Participants will receive placebo.
Drug: Placebo
Placebo will be administered via IV infusion once a week for 4 weeks.




Primary Outcome Measures :
  1. Time To First Reaching A Hughes FG Score ≤ 1 [ Time Frame: Up to Week 24 ]

Secondary Outcome Measures :
  1. Proportion Of Participants With A Hughes FG Score ≤ 1 [ Time Frame: Week 24 ]
  2. Proportion Of Participants With A Hughes FG Score Improvement Of ≥ 3 [ Time Frame: Week 24 ]
  3. Proportion Of Participants With A Hughes FG Score ≤ 1 [ Time Frame: Week 8 ]
  4. Incidence Of Treatment-emergent Adverse Events [ Time Frame: Up to Week 24 ]
  5. Free Complement Component 5 In Serum [ Time Frame: Week 24 ]
  6. Hemolytic Complement Activity In Serum [ Time Frame: Week 24 ]
  7. Length Of Stay In The Hospital [ Time Frame: Up to Week 24 ]
  8. Duration Of Ventilator Support [ Time Frame: Up to Week 24 ]
  9. Concentration Of Eculizumab In Serum [ Time Frame: Up to Week 24 ]
  10. Incidence Of Antidrug Antibodies [ Time Frame: Up to Week 24 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04752566


Locations
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Japan
Clinical Trial Site
Bunkyo-ku, Japan, 113-8519
Clinical Trial Site
Chiba, Japan, 260-8677
Clinical Trial Site
Fuchu, Japan, 183-0042
Clinical Trial Site
Fukuoka, Japan, 814-0180
Clinical Trial Site
Gifu, Japan, 501-1194
Clinical Trial Site
Hiroshima, Japan, 730-8518
Clinical Trial Site
Kagoshima, Japan, 890-8520
Clinical Trial Site
Kawagoe, Japan, 350-8550
Clinical Trial Site
Kawasaki, Japan, 216-8511
Clinical Trial Site
Kitakyushu, Japan, 807-8556
Clinical Trial Site
Kobe, Japan, 650-0047
Clinical Trial Site
Kumamoto, Japan, 860-8556
Clinical Trial Site
Kurashiki, Japan, 710-8602
Clinical Trial Site
Kyoto, Japan, 602-8566
Clinical Trial Site
Matsumoto, Japan, 390-8621
Clinical Trial Site
Mibu, Japan, 321-0293
Clinical Trial Site
Mitaka, Japan, 181-8611
Clinical Trial Site
Nagoya, Japan, 466-8560
Clinical Trial Site
Niigata, Japan, 951-8520
Clinical Trial Site
Nishinomiya, Japan, 663-8501
Clinical Trial Site
Osakasayama, Japan, 589-8511
Clinical Trial Site
Sagamihara, Japan, 252-0375
Clinical Trial Site
Sapporo, Japan, 060-8648
Clinical Trial Site
Sendai, Japan, 983-8520
Clinical Trial Site
Ube, Japan, 755-8505
Clinical Trial Site
Yokohama, Japan, 236-0004
Sponsors and Collaborators
Alexion Pharmaceuticals
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04752566    
Other Study ID Numbers: ECU-GBS-301
First Posted: February 12, 2021    Key Record Dates
Last Update Posted: April 12, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion Pharmaceuticals:
Eculizumab
Soliris
Alexion
C5 Inhibition Therapy
Additional relevant MeSH terms:
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Eculizumab
Guillain-Barre Syndrome
Syndrome
Disease
Pathologic Processes
Polyradiculoneuropathy
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Peripheral Nervous System Diseases
Neuromuscular Diseases
Polyneuropathies
Autoimmune Diseases
Immune System Diseases
Complement Inactivating Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs