Clinical Trial Results:
Inhibition of complement activation (eculizumab®) in Guillain-Barré Syndrome study
Summary
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EudraCT number |
2013-000228-33 |
Trial protocol |
GB |
Global end of trial date |
30 Jul 2016
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Results information
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Results version number |
v1(current) |
This version publication date |
27 Feb 2020
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First version publication date |
27 Feb 2020
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Other versions |
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
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Trial identification
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Sponsor protocol code |
GN12NE462
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Additional study identifiers
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ISRCTN number |
- | ||
US NCT number |
NCT02029378 | ||
WHO universal trial number (UTN) |
- | ||
Sponsors
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Sponsor organisation name |
University of Glasgow
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Sponsor organisation address |
126 University Place, Glasgow, United Kingdom, G12 8TA
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Public contact |
Prof. Hugh J. Willison, University of Glasgow, 0044 141 330 8384, Hugh.Willison@glasgow.ac.uk
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Scientific contact |
Prof. Hugh J. Willison, University of Glasgow, 0044 141 330 8384, Hugh.Willison@glasgow.ac.uk
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Sponsor organisation name |
NHS Greater Glasgow and Clyde
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Sponsor organisation address |
Grahamston Road, Paisley, United Kingdom, PA2 7DE
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Public contact |
Maureen Travers, NHS Greater Glasgow and Clyde, Maureen.Travers@ggc.scot.nhs.uk
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Scientific contact |
Maureen Travers, NHS Greater Glasgow and Clyde, Maureen.Travers@ggc.scot.nhs.uk
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Paediatric regulatory details
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Is trial part of an agreed paediatric investigation plan (PIP) |
No
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Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
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Results analysis stage
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Analysis stage |
Final
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Date of interim/final analysis |
30 Jul 2016
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Is this the analysis of the primary completion data? |
Yes
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Primary completion date |
30 Jul 2016
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Global end of trial reached? |
Yes
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Global end of trial date |
30 Jul 2016
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Was the trial ended prematurely? |
Yes
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General information about the trial
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Main objective of the trial |
To evaluate safety and tolerability, of eculizumab in patients with GBS.
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Protection of trial subjects |
All the patients with GBS receive standard treatment either IVIg or Plasma exchange. Only those patients who would be eligible for IVIg treatment will be screened for the study. Patient’s participation will not delay the commencement of standard IVIg treatment. All the patients will be recruited within 14 days of onset of weakness, provided that the study drug can be started within this 14 day time period.
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Background therapy |
All participants received ciprofloxacin as a prophylactic antibiotic for 10 weeks. | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
01 Sep 2013
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Long term follow-up planned |
No
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Independent data monitoring committee (IDMC) involvement? |
Yes
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Population of trial subjects
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Number of subjects enrolled per country |
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Country: Number of subjects enrolled |
United Kingdom: 8
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Worldwide total number of subjects |
8
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EEA total number of subjects |
8
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Number of subjects enrolled per age group |
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In utero |
0
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Preterm newborn - gestational age < 37 wk |
0
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Newborns (0-27 days) |
0
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Infants and toddlers (28 days-23 months) |
0
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Children (2-11 years) |
0
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Adolescents (12-17 years) |
0
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Adults (18-64 years) |
6
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From 65 to 84 years |
2
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85 years and over |
0
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Recruitment
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Recruitment details |
Participants recruited between Aug 2014 to Jul 2016. | |||||||||||||||
Pre-assignment
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Screening details |
All patients diagnosed with GBS and unable to walk within 2 weeks were screened. | |||||||||||||||
Period 1
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Period 1 title |
Baseline (overall period)
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Is this the baseline period? |
Yes | |||||||||||||||
Allocation method |
Randomised - controlled
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Blinding used |
Double blind | |||||||||||||||
Roles blinded |
Subject, Investigator, Data analyst, Carer | |||||||||||||||
Arms
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Are arms mutually exclusive |
Yes
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Arm title
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Eculizumab | |||||||||||||||
Arm description |
Following consent, participants will be randomly assigned in a ratio of 2:1 to receive either Eculizumab or matching placebo by contacting the interactive webresponse system (IWRS). Drug treatment will be commenced as soon as is practical, after consent is obtained. | |||||||||||||||
Arm type |
Experimental | |||||||||||||||
Investigational medicinal product name |
Eculizumab
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Powder and solvent for solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
administered intravenously at a dose of 900mg weekly for 4 weeks (day 0, week 1, week 2 and week 3)
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Arm title
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Placebo | |||||||||||||||
Arm description |
Following consent, participants will be randomly assigned in a ratio of 2:1 to receive either Eculizumab or matching placebo by contacting the interactive webresponse system (IWRS). Drug treatment will be commenced as soon as is practical, after consent is obtained | |||||||||||||||
Arm type |
Placebo | |||||||||||||||
Investigational medicinal product name |
Placebo
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Investigational medicinal product code |
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Other name |
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Pharmaceutical forms |
Powder and solvent for concentrate for solution for infusion
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Routes of administration |
Intravenous use
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Dosage and administration details |
administered intravenously at a dose of 900mg weekly for 4 weeks (day 0, week 1, week 2 and week 3)
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Baseline characteristics reporting groups
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Reporting group title |
Baseline
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||
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End points reporting groups
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Reporting group title |
Eculizumab
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Reporting group description |
Following consent, participants will be randomly assigned in a ratio of 2:1 to receive either Eculizumab or matching placebo by contacting the interactive webresponse system (IWRS). Drug treatment will be commenced as soon as is practical, after consent is obtained. | ||
Reporting group title |
Placebo
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Reporting group description |
Following consent, participants will be randomly assigned in a ratio of 2:1 to receive either Eculizumab or matching placebo by contacting the interactive webresponse system (IWRS). Drug treatment will be commenced as soon as is practical, after consent is obtained |
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End point title |
Functional outcome on the GBS disability scale at set time intervals [1] | |||||||||
End point description |
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End point type |
Primary
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End point timeframe |
Assessed from week 0 to week 4
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Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: Because of the relevantly small number of participants and the primary end endpoint being an improvement in a scale, there was no statistical analysis formed and the improvement in the GBS scale for each participant simply reported. |
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No statistical analyses for this end point |
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Adverse events information
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Timeframe for reporting adverse events |
From time of consent to 30 days following the last study visit.
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Assessment type |
Non-systematic | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary used for adverse event reporting
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Dictionary name |
MedDRA | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
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Reporting groups
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Reporting group title |
Eculizaumab
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Reporting group description |
- | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Placebo
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Reporting group description |
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Frequency threshold for reporting non-serious adverse events: 0% | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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Substantial protocol amendments (globally) |
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Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
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08 Jan 2016 |
Protocol change and updated patient alert card |
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Interruptions (globally) |
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Were there any global interruptions to the trial? No | |||
Limitations and caveats |
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Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
None reported | |||
Online references |
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http://www.ncbi.nlm.nih.gov/pubmed/27801990 |