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

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ClinicalTrials.gov Identifier: NCT04752566
Recruitment Status : Completed
First Posted : February 12, 2021
Results First Posted : February 9, 2024
Last Update Posted : February 9, 2024
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Guillain-Barre Syndrome
Interventions Biological: Eculizumab
Drug: Placebo
Enrollment 57
Recruitment Details  
Pre-assignment Details All participants were on concomitant intravenous immunoglobulin (IVIg) therapy as per standard of care.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description Participants received eculizumab intravenous (IV) infusion on Days 1, 8, 15, and 22. Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Period Title: Overall Study
Started 37 20
Received at Least 1 Dose of Study Drug 37 20
Completed 32 18
Not Completed 5 2
Reason Not Completed
Protocol Violation             0             1
Death             1             0
Adverse Event             1             0
Withdrawal by Subject             2             0
Physician Decision             1             1
Arm/Group Title Eculizumab Placebo Total
Hide Arm/Group Description Participants received eculizumab IV infusion on Days 1, 8, 15, and 22. Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22. Total of all reporting groups
Overall Number of Baseline Participants 37 20 57
Hide Baseline Analysis Population Description
Full analysis set included all randomized participants who received at least 1 dose of study drug and had a baseline functional grade (FG) score and at least 1 postbaseline FG score.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 37 participants 20 participants 57 participants
56.6  (19.61) 56.2  (18.34) 56.4  (19.01)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 37 participants 20 participants 57 participants
Female
19
  51.4%
5
  25.0%
24
  42.1%
Male
18
  48.6%
15
  75.0%
33
  57.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 37 participants 20 participants 57 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
37
 100.0%
20
 100.0%
57
 100.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 37 participants 20 participants 57 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
37
 100.0%
20
 100.0%
57
 100.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
0
   0.0%
0
   0.0%
0
   0.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Time to First Reaching a Hughes Functional Grade (FG) Score <=1
Hide Description The mobility of the participants was evaluated on a 7 point disability functional grade scale and described as Hughes FG score of 0 (Healthy, no signs or symptoms of Guillain-Barré syndrome); 1 (Minor signs or symptoms and able to run); 2 (Able to walk 5 metre (m) across an open space without assistance); 3 (Able to walk 5 m across an open space with the help of one person and waist-level walking-frame, stick, or sticks); 4 (Chairbound/bedbound: unable to walk as in 3); 5 (Requiring assisted ventilation [for at least part of day or night]) and 6 (Dead), where higher numbers indicate more severe impairment. The Kaplan-Meier estimate of time to event of FG<=1 is reported. Time (days) to first event=Date of first event-Date of first dose+1. Participants who discontinued early without achieving FG <= 1 were censored at the date of discontinuation. Participants who completed the study without achieving FG<=1 were censored at the date of study completion.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set included all randomized participants who received at least 1 dose of study drug and had a baseline FG score and at least 1 postbaseline FG score. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 20 13
Median (95% Confidence Interval)
Unit of Measure: Days
112.0 [1] 
(59.0 to NA)
168.0 [1] 
(36.0 to NA)
[1]
NA signifies upper limit was not estimable as study does not have enough events to estimate the median time to event.
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8938
Comments Log Rank Test stratified by randomization strata.
Method Log Rank
Comments [Not Specified]
Method of Estimation Estimation Parameter Hazard Ratio (HR)
Estimated Value 0.9
Confidence Interval (2-Sided) 95%
0.45 to 1.97
Estimation Comments Cox proportional hazard model stratified by randomization strata, with treatment group as the fixed effect. Firth's adjustment was applied if no event was observed in a treatment group.
2.Secondary Outcome
Title Number of Participants With A Hughes Functional Grade (FG) Score <=1
Hide Description The mobility of the participants was evaluated on a 7 point disability FG scale and described as Hughes FG score of 0 (Healthy, no signs or symptoms of Guillain-Barré syndrome); 1 (Minor signs or symptoms and able to run); 2 (Able to walk 5 m across an open space without assistance); 3 (Able to walk 5 m across an open space with the help of one person and waist-level walking-frame, stick, or sticks); 4 (Chairbound/bedbound: unable to walk as in 3); 5 (Requiring assisted ventilation [for at least part of day or night]) and 6 (Dead), where higher numbers indicate more severe impairment. If a participant had an FG score <= 1 prior to or at Week 8 and Week 24, respectively, then the participant is considered a responder. Otherwise, participants discontinued prior to Week 8 and Week 24 or with an FG score > 1 at Week 8 and Week 24 are nonresponders, respectively.
Time Frame Week 8, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set included all randomized participants who received at least 1 dose of study drug and had a baseline FG score and at least 1 postbaseline FG score.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 37 20
Measure Type: Count of Participants
Unit of Measure: Participants
Week 8
12
  32.4%
7
  35.0%
Week 24
20
  54.1%
13
  65.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab, Placebo
Comments Week 8
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8987
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.9
Confidence Interval (2-Sided) 95%
0.27 to 3.17
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Eculizumab, Placebo
Comments Week 24
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4083
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.6
Confidence Interval (2-Sided) 95%
0.18 to 2.02
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Number of Participants With A Hughes Functional Grade Score Improvement of >=3
Hide Description The mobility of the participants was evaluated on a 7 point disability FG scale and described as Hughes FG score of 0 (Healthy, no signs or symptoms of Guillain-Barré syndrome); 1 (Minor signs or symptoms and able to run); 2 (Able to walk 5 m across an open space without assistance); 3 (Able to walk 5 m across an open space with the help of one person and waist-level walking-frame, stick, or sticks); 4 (Chairbound/bedbound: unable to walk as in 3); 5 (Requiring assisted ventilation [for at least part of day or night]) and 6 (Dead), where higher numbers indicate more severe impairment. Participants with a change from baseline in FG score (value at Week 24 - baseline value) <= -3 were considered a responder. Participants with change from baseline > -3 and participants who discontinued prior to Week 24 were considered non-responders.
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set included all randomized participants who received at least 1 dose of study drug and had a baseline FG score and at least 1 postbaseline FG score.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 37 20
Measure Type: Count of Participants
Unit of Measure: Participants
16
  43.2%
10
  50.0%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Eculizumab, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6739
Comments [Not Specified]
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.8
Confidence Interval (2-Sided) 95%
0.24 to 2.54
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Hide Description TEAEs were defined as an adverse event (AE) with onset on or after the first dose of the study drug. An AE is any untoward medical occurrence in a participant, temporally associated with the use of study drug, whether or not considered related to the study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Time Frame Day 1 up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all participants who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 37 20
Measure Type: Count of Participants
Unit of Measure: Participants
34
  91.9%
19
  95.0%
5.Secondary Outcome
Title Free Complement Component 5 in Serum
Hide Description [Not Specified]
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic (PD) analysis set included all participants who received at least 1 dose of study drug and who have at least 1 postdose PD sample. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 32 18
Mean (Standard Deviation)
Unit of Measure: micrograms/milliliters
131.0438  (29.40068) 147.8833  (36.70182)
6.Secondary Outcome
Title Hemolytic Complement Activity in Serum
Hide Description [Not Specified]
Time Frame Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamic analysis set included all participants who received at least 1 dose of study drug and who have at least 1 postdose PD sample. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 32 18
Mean (Standard Deviation)
Unit of Measure: percentage of hemolysis
91.79  (11.077) 91.83  (11.767)
7.Secondary Outcome
Title Length of Stay in the Hospital
Hide Description For participants with multiple hospitalizations, the total duration of all hospitalizations was summarized.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set included all randomized participants who received at least 1 dose of study drug and had a baseline FG score and at least 1 postbaseline FG score.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 37 20
Mean (Standard Deviation)
Unit of Measure: days
49.5  (27.15) 39.8  (12.97)
8.Secondary Outcome
Title Number of Participants Who Required Mechanical Ventilator Support
Hide Description For participants with more than 1 episode of the same support, the total duration across all episodes was summarized.
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Full analysis set included all randomized participants who received at least 1 dose of study drug and had a baseline FG score and at least 1 postbaseline FG score.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 37 20
Measure Type: Count of Participants
Unit of Measure: Participants
8
  21.6%
1
   5.0%
9.Secondary Outcome
Title Concentration of Eculizumab in Serum
Hide Description [Not Specified]
Time Frame Up to Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic analysis set (PKAS) included all participants who received at least 1 dose of study drug and who had at least 1 postdose PK sample. Here, Number of Participants analyzed signifies those who were evaluable for this outcome measure.
Arm/Group Title Eculizumab
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 32
Mean (Standard Deviation)
Unit of Measure: micrograms/milliliters
4.690  (0.0000)
10.Secondary Outcome
Title Number of Participants With Positive Antidrug Antibodies
Hide Description [Not Specified]
Time Frame Up to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all participants who received at least 1 dose of study drug.
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description:
Participants received eculizumab IV infusion on Days 1, 8, 15, and 22.
Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
Overall Number of Participants Analyzed 37 20
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
Time Frame Day 1 up to Week 24
Adverse Event Reporting Description Safety set included all participants who received at least 1 dose of study drug.
 
Arm/Group Title Eculizumab Placebo
Hide Arm/Group Description Participants received eculizumab IV infusion on Days 1, 8, 15, and 22. Participants received placebo matched to eculizumab via IV infusion on Days 1, 8, 15, and 22.
All-Cause Mortality
Eculizumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   1/37 (2.70%)   0/20 (0.00%) 
Hide Serious Adverse Events
Eculizumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   4/37 (10.81%)   1/20 (5.00%) 
General disorders     
Hanging * 1  1/37 (2.70%)  0/20 (0.00%) 
Infections and infestations     
Pneumonia aspiration * 1  2/37 (5.41%)  0/20 (0.00%) 
COVID-19 * 1  1/37 (2.70%)  0/20 (0.00%) 
Nervous system disorders     
Chronic inflammatory demyelinating polyradiculoneuropathy * 1  0/37 (0.00%)  1/20 (5.00%) 
1
Term from vocabulary, MedDRA v25.0
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Eculizumab Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   34/37 (91.89%)   18/20 (90.00%) 
Blood and lymphatic system disorders     
Anaemia * 1  4/37 (10.81%)  0/20 (0.00%) 
Leukopenia * 1  0/37 (0.00%)  1/20 (5.00%) 
Cardiac disorders     
Bradycardia * 1  0/37 (0.00%)  1/20 (5.00%) 
Eye disorders     
Dry eye * 1  2/37 (5.41%)  0/20 (0.00%) 
Gastrointestinal disorders     
Constipation * 1  4/37 (10.81%)  7/20 (35.00%) 
Diarrhoea * 1  5/37 (13.51%)  1/20 (5.00%) 
Stomatitis * 1  2/37 (5.41%)  1/20 (5.00%) 
Abdominal distension * 1  2/37 (5.41%)  0/20 (0.00%) 
Gastritis * 1  2/37 (5.41%)  0/20 (0.00%) 
Vomiting * 1  2/37 (5.41%)  0/20 (0.00%) 
General disorders     
Pyrexia * 1  5/37 (13.51%)  0/20 (0.00%) 
Oedema * 1  0/37 (0.00%)  1/20 (5.00%) 
Pain * 1  0/37 (0.00%)  1/20 (5.00%) 
Hepatobiliary disorders     
Hepatic function abnormal * 1  4/37 (10.81%)  2/20 (10.00%) 
Drug-induced liver injury * 1  3/37 (8.11%)  0/20 (0.00%) 
Liver disorder * 1  0/37 (0.00%)  1/20 (5.00%) 
Immune system disorders     
Hypersensitivity * 1  0/37 (0.00%)  1/20 (5.00%) 
Infections and infestations     
Urinary tract infection * 1  5/37 (13.51%)  3/20 (15.00%) 
Pneumonia aspiration * 1  5/37 (13.51%)  2/20 (10.00%) 
COVID-19 * 1  0/37 (0.00%)  1/20 (5.00%) 
Conjunctivitis * 1  0/37 (0.00%)  1/20 (5.00%) 
Genital candidiasis * 1  0/37 (0.00%)  1/20 (5.00%) 
Injury, poisoning and procedural complications     
Skin abrasion * 1  0/37 (0.00%)  1/20 (5.00%) 
Investigations     
Aspartate aminotransferase increased * 1  2/37 (5.41%)  3/20 (15.00%) 
Alanine aminotransferase increased * 1  1/37 (2.70%)  3/20 (15.00%) 
Liver function test increased * 1  1/37 (2.70%)  2/20 (10.00%) 
Neutrophil count decreased * 1  2/37 (5.41%)  0/20 (0.00%) 
Blood creatine phosphokinase increased * 1  1/37 (2.70%)  1/20 (5.00%) 
Blood lactate dehydrogenase increased * 1  0/37 (0.00%)  1/20 (5.00%) 
C-reactive protein increased * 1  0/37 (0.00%)  1/20 (5.00%) 
Fibrin D dimer increased * 1  0/37 (0.00%)  1/20 (5.00%) 
Gamma-glutamyltransferase increased * 1  0/37 (0.00%)  1/20 (5.00%) 
Protein urine * 1  0/37 (0.00%)  1/20 (5.00%) 
Metabolism and nutrition disorders     
Hypokalaemia * 1  5/37 (13.51%)  1/20 (5.00%) 
Hyponatraemia * 1  0/37 (0.00%)  3/20 (15.00%) 
Decreased appetite * 1  2/37 (5.41%)  0/20 (0.00%) 
Dehydration * 1  1/37 (2.70%)  1/20 (5.00%) 
Musculoskeletal and connective tissue disorders     
Back pain * 1  2/37 (5.41%)  1/20 (5.00%) 
Myalgia * 1  3/37 (8.11%)  0/20 (0.00%) 
Neck pain * 1  2/37 (5.41%)  0/20 (0.00%) 
Musculoskeletal stiffness * 1  0/37 (0.00%)  1/20 (5.00%) 
Nervous system disorders     
Headache * 1  5/37 (13.51%)  3/20 (15.00%) 
Neuropathy peripheral * 1  1/37 (2.70%)  1/20 (5.00%) 
Facial paralysis * 1  0/37 (0.00%)  1/20 (5.00%) 
Lumbar radiculopathy * 1  0/37 (0.00%)  1/20 (5.00%) 
Restless legs syndrome * 1  0/37 (0.00%)  1/20 (5.00%) 
Psychiatric disorders     
Insomnia * 1  10/37 (27.03%)  8/20 (40.00%) 
Delirium * 1  2/37 (5.41%)  0/20 (0.00%) 
Renal and urinary disorders     
Dysuria * 1  1/37 (2.70%)  1/20 (5.00%) 
Haematuria * 1  2/37 (5.41%)  0/20 (0.00%) 
Renal impairment * 1  0/37 (0.00%)  1/20 (5.00%) 
Reproductive system and breast disorders     
Genital rash * 1  0/37 (0.00%)  1/20 (5.00%) 
Respiratory, thoracic and mediastinal disorders     
Sputum retention * 1  0/37 (0.00%)  1/20 (5.00%) 
Skin and subcutaneous tissue disorders     
Dermatitis contact * 1  3/37 (8.11%)  3/20 (15.00%) 
Rash * 1  4/37 (10.81%)  1/20 (5.00%) 
Dry skin * 1  4/37 (10.81%)  0/20 (0.00%) 
Eczema * 1  2/37 (5.41%)  1/20 (5.00%) 
Dermatitis diaper * 1  1/37 (2.70%)  1/20 (5.00%) 
Skin erosion * 1  1/37 (2.70%)  1/20 (5.00%) 
Asteatosis * 1  0/37 (0.00%)  1/20 (5.00%) 
Eczema asteatotic * 1  0/37 (0.00%)  1/20 (5.00%) 
Rash maculo-papular * 1  0/37 (0.00%)  1/20 (5.00%) 
Vascular disorders     
Deep vein thrombosis * 1  4/37 (10.81%)  1/20 (5.00%) 
Venous thrombosis limb * 1  1/37 (2.70%)  1/20 (5.00%) 
Hypotension * 1  0/37 (0.00%)  1/20 (5.00%) 
1
Term from vocabulary, MedDRA v25.0
*
Indicates events were collected by non-systematic assessment
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Alexion Pharmaceuticals Inc.
Organization: Alexion Pharmaceuticals Inc.
Phone: +1 855-752-2356
EMail: clinicaltrials@alexion.com
Layout table for additonal information
Responsible Party: Alexion Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04752566    
Other Study ID Numbers: ECU-GBS-301
First Submitted: February 9, 2021
First Posted: February 12, 2021
Results First Submitted: June 6, 2023
Results First Posted: February 9, 2024
Last Update Posted: February 9, 2024