Trial · NCT04752566

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

NCT04752566, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2024-02-09

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome. Trials, record NCT04752566. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04752566

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study to Evaluate the Efficacy and Safety of Eculizumab in Guillain-Barré Syndrome
NCT number: NCT04752566
Status: Completed
Phases: Phase 3
Enrollment: 57 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Guillain-Barre Syndrome
Interventions: Eculizumab, Placebo
First posted: 2021-02-12
Last updated: 2025-09-08
Results first posted: 2024-02-09

Source ClinicalTrials.gov

Registered outcomes10

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Time to First Reaching a Hughes Functional Grade (FG) Score <=1	Up to Week 24	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.
Secondary	Number of Participants With A Hughes Functional Grade (FG) Score <=1	Week 8, Week 24	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.
Secondary	Number of Participants With A Hughes Functional Grade Score Improvement of >=3	Week 24	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.
Secondary	Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Day 1 up to Week 24	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.
Secondary	Free Complement Component 5 in Serum	Week 24
Secondary	Hemolytic Complement Activity in Serum	Week 24
Secondary	Length of Stay in the Hospital	Up to Week 24	For participants with multiple hospitalizations, the total duration of all hospitalizations was summarized.
Secondary	Number of Participants Who Required Mechanical Ventilator Support	Up to Week 24	For participants with more than 1 episode of the same support, the total duration across all episodes was summarized.
Secondary	Concentration of Eculizumab in Serum	Up to Week 24
Secondary	Number of Participants With Positive Antidrug Antibodies	Up to Week 12

Source ClinicalTrials.gov

Outcomes10

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Time to First Reaching a Hughes Functional Grade (FG) Score <=1	Up to Week 24	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.	Days	MEDIAN
SECONDARY	Number of Participants With A Hughes Functional Grade (FG) Score <=1	Week 8, Week 24	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.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Number of Participants With A Hughes Functional Grade Score Improvement of >=3	Week 24	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.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Day 1 up to Week 24	Safety set included all participants who received at least 1 dose of study drug.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Free Complement Component 5 in Serum	Week 24	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.	micrograms/milliliters	MEAN
SECONDARY	Hemolytic Complement Activity in Serum	Week 24	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.	percentage of hemolysis	MEAN
SECONDARY	Length of Stay in the Hospital	Up to Week 24	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.	days	MEAN
SECONDARY	Number of Participants Who Required Mechanical Ventilator Support	Up to Week 24	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.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Concentration of Eculizumab in Serum	Up to Week 24	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.	micrograms/milliliters	MEAN
SECONDARY	Number of Participants With Positive Antidrug Antibodies	Up to Week 12	Safety set included all participants who received at least 1 dose of study drug.	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group21

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Time to First Reaching a Hughes Functional Grade (FG) Score <=1	Eculizumab		112.0	95% Confidence Interval 59.0 to NA	Days
Time to First Reaching a Hughes Functional Grade (FG) Score <=1	Placebo		168.0	95% Confidence Interval 36.0 to NA	Days
Number of Participants With A Hughes Functional Grade (FG) Score <=1	Eculizumab	Week 8	12		Participants
Number of Participants With A Hughes Functional Grade (FG) Score <=1	Placebo	Week 8	7		Participants
Number of Participants With A Hughes Functional Grade (FG) Score <=1	Eculizumab	Week 24	20		Participants
Number of Participants With A Hughes Functional Grade (FG) Score <=1	Placebo	Week 24	13		Participants
Number of Participants With A Hughes Functional Grade Score Improvement of >=3	Eculizumab		16		Participants
Number of Participants With A Hughes Functional Grade Score Improvement of >=3	Placebo		10		Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Eculizumab		34		Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Placebo		19		Participants

1 to 10 of 21Source ClinicalTrials.gov

See all 21 results by group

Participant flow8

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Eculizumab	37
Overall Study	STARTED	Placebo	20
Overall Study	Received at Least 1 Dose of Study Drug	Eculizumab	37
Overall Study	Received at Least 1 Dose of Study Drug	Placebo	20
Overall Study	COMPLETED	Eculizumab	32
Overall Study	COMPLETED	Placebo	18
Overall Study	NOT COMPLETED	Eculizumab	5
Overall Study	NOT COMPLETED	Placebo	2

Source ClinicalTrials.gov

Adverse events138

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Eculizumab	4	37
other	Anaemia	Blood and lymphatic system disorders	Placebo	0	20
other	Leukopenia	Blood and lymphatic system disorders	Eculizumab	0	37
other	Leukopenia	Blood and lymphatic system disorders	Placebo	1	20
other	Bradycardia	Cardiac disorders	Eculizumab	0	37
other	Bradycardia	Cardiac disorders	Placebo	1	20
other	Dry eye	Eye disorders	Eculizumab	2	37
other	Dry eye	Eye disorders	Placebo	0	20
other	Constipation	Gastrointestinal disorders	Eculizumab	4	37
other	Constipation	Gastrointestinal disorders	Placebo	7	20

1 to 10 of 138Source ClinicalTrials.gov

See all 138 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Eculizumab	1	37
Total, all-cause mortality	Placebo	0	20
Total, other adverse events	Eculizumab	34	37
Total, other adverse events	Placebo	18	20
Total, serious adverse events	Eculizumab	4	37
Total, serious adverse events	Placebo	1	20

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
38987228	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons6

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Eculizumab vs Placebo: Number of Participants With Treatment-emergent Adverse Events (TEAEs)	Trial comparisons
Eculizumab vs Placebo: Number of Participants With A Hughes Functional Grade Score Improvement of >=3	Trial comparisons
Eculizumab vs Placebo: Number of Participants Who Required Mechanical Ventilator Support	Trial comparisons
Eculizumab vs Placebo: Number of Participants With Positive Antidrug Antibodies	Trial comparisons
Eculizumab vs Placebo: deaths from any cause	Trial comparisons
Eculizumab vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT04752566

GET https://conductscience.org/api/trials/v1/NCT04752566/results?page=2

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