Gullian Barre SyndromePhase 2ICA-GBSNCT02029378
Inhibition of Complement Activation (Eculizumab) in Guillain-Barre Syndrome Study
A Phase 2 study testing Eculizumab in people with Gullian Barre Syndrome, run by NHS Greater Glasgow and Clyde at 1 site in United Kingdom.
Trial summary
Guillian-Barre Syndrome (GBS) is the most frequent cause of acute neuromuscular weakness in the Western World and can occur at any age. GBS is a rpadily progressive 'inflammatory' disorder of the perihperal nerves often leading to sever paresis of the limbs. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gullian Barre Syndrome
- What is being tested?
- Eculizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Sep 2014to Mar 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients aged >18 years diagnosed with GBS according to NINDS diagnostic criteria
- Onset of weakness due to GBS is less than 2 weeks ago
- Patients who are unable to walk unaided for >10 metres (grade >3 on GBS disability scale)
- Patients who are being considered for or already on IVIg treatment
- First dose of eculizumab must be started within 2 weeks from onset of weakness and any time during the IVIg treatment period
- Signed informed consent
Who cannot join
- Age <18 years
- Patients who are being considered for, or already on, plasma exchange
- Pregnancy or lactation
- Patients show clear clinical evidence of a polyneuropahty caused by e.g. diabetes mellitus (except mild sensory), alcoholism, severe vitamin deficiency, and porphyria
- Patients received immunosuppressive treatment (e.g. azathioprine, cyclosporine, mycofenolatemofetil, tacrolimus, sirolimus or > 20 mg prednisolone daily) during the last month
- Patients known to have severe concurrent disease, like malignancy, severe cardiovascular disease, AIDS, severe COPD, TB
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- Inability to comply with study related procedures or appointments during 6 months
- Any condition that in the opinion of the investigator could increase the patient's risk by participating in the study or confound the outcome of the study
- Related to the administration of eculizumab:
- Unresoled Neisseria meningitidisinfection of history of meningococcal infection Unsuitable for antibiotic prophylaxis (e.g due to allergy) Known hypersensitivity to eculizumab, murine proteins or to any of the excipients Known or suspected hereditary complement deficiencies Women of child-bearing potential who are unwilling to use effective contraception during treatment and for 5 months after treatment is completed.
Trial locations and contacts
1 site in 1 country.
Govindsinh Chavada contact
United Kingdom1 site, 1 recruiting
- Recruiting
Southern General Hospital
Glasgow, G51 4TF
Govindsinh Chavada, govindsinh.chavada@glasgow.ac.uk
Amy Davidson, MBChB, BSc, MRCP, amy.davidson2@nhs.net
About this study
Guillian-Barre Syndrome (GBS) is the most frequent cause of acute neuromuscular weakness in the Western World and can occur at any age. GBS is a rpadily progressive 'inflammatory' disorder of the perihperal nerves often leading to sever paresis of the limbs. Most GBS patients also have sensory disturbances (tingling or dull feeling) and pain. Some patients also have double vision or problems with swallowing. GBS mau also involve the respiratory muscles, leading to insufficient ventilation and admission to an intensive care unit. GBS pateints have a vairable prognosis; 20-30% require mechnical ventilation for a period ranging from weeks to months, 20% are unable to walk after 6 months nad 3-5% dies. Progression of weakness in GBS is usually rapid and reaches its peak within 4 weeks in the majority of patients, but many develop their maximum deficit within 2 weeks. Thereafter, the patients have a variable prognosis.
GBS is a treatable disorder. Intravenous immunoglobulin (IVIg) 2g/kg administered in 5 days was shown to be effective when administered within the first two weeks after onset of symptoms, and is considered the treatment of choice by most experts in the field. Although the standard treatment for GBS is a single course of IVIg (2g/kg administered in 5 days), many patients fails to recover abd remain with substantial disability. Patients with GBS and especially those with a poor prognosis potentially may benefit from more powerful abd when possible a more mechanistically rational therapy.
Recent experimental evidence suggests that complement activation palys a crucial role in the development of neuromuscular weakness in GBS making complement inhibitors and regulators attracive therapeutic targets. Our hypothesis is that Eculizumab, with its function as a complement inhibitor, will be very effective in preventing progression of weakness in patients with GBS.
- Conditions
- Gullian Barre Syndrome
- Being tested
- Eculizumab
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EculizumabDrug
| Study group | Role | What they receive |
|---|---|---|
| Eculizumab | Experimental | Eculizumab, 900 mg intravenously once a week Receives: Drug: Eculizumab |
| Placebo | Placebo comparator | Matched placebo, intravenously once a week |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the incidence of AE/SAEs after treatment with eculizumab and IVIg compared to placebo controls
Primary safety endpoint
Measured: 6 months
Improvement of one or more grade in functional outcome (on the 6 point GBS disability scale) at 4 weeks
Primary efficacy endpoint
Measured: 4 weeks
Secondary outcomes (10)
Ability to walk unaided (GBS disability score 2) at 8 weeks
Measured: 8 weeks
Time taken to improve by at least one grade (on the GBS disability scale)
Measured: 8 weeks
Time taken to walk independently
Measured: 1 year
Difference in GBS disability score at maximum disability completed with 6 months
Measured: 6 months
Percentage of patients with a clinically relevant improvement in R-ODS score
An increase from Baseline in R-ODS score by at least 6 points on the centile metric score at 4 weeks and 6 months
Measured: 6 months
Percentage of patients with a clinically relevant improvement in ONLS
Defined as an increase from baseline in ONLS score by at least 1 point at 4 weeks and 6 months
Measured: 6 months
Requirement for ventilatory support (GBS disability score 5)
Measured: 4 weeks
Duration of ventilatory support
Measured: 8 weeks
Occurrence of relapse
Measured: 2 years
Dearth within the first 6 months
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2014 (estimated)
- Study startSep 2014
- Primary completionOct 2015 (estimated)
- Study completionMar 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Gullian Barre SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- NHS Greater Glasgow and Clyde
- Sponsor type
- University, hospital or other
- Collaborators
- University of Glasgow
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02029378
- Sponsor's protocol ID
- GN12NE462
- Acronym
- ICA-GBS
- Status last verified
- Sep 2014
- First submitted
- 6 Jan 2014
- Last updated
- 23 Sep 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Davidson AI, Halstead SK, Goodfellow JA, Chavada G, Mallik A, Overell J, Lunn MP, McConnachie A, van Doorn P, Willison HJ. Inhibition of complement in Guillain-Barre syndrome: the ICA-GBS study. J Peripher Nerv Syst. 2017 Mar;22(1):4-12. doi: 10.1111/jns.12194.
PubMed 27801990
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Inhibition of Complement Activation (Eculizumab) in Guillain-Barre Syndrome Study
- ClinicalTrials.gov ID
- NCT02029378
- Acronym / sponsor ID
- ICA-GBS / GN12NE462
- Conditions
- Gullian Barre Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- NHS Greater Glasgow and ClydeUniversity, hospital or other
- Collaborators
- University of Glasgow
- Study start
- Sep 2014
- Primary completion
- Oct 2015 (estimated)
- Study completion
- Mar 2016 (estimated)
- First posted
- 7 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Guillian-Barre Syndrome (GBS) is the most frequent cause of acute neuromuscular weakness in the Western World and can occur at any age. GBS is a rpadily progressive 'inflammatory' disorder of the perihperal nerves often leading to sever paresis of the limbs. Most GBS patients also have sensory disturbances (tingling or dull feeling) and pain. Some patients also have double vision or problems with swallowing. GBS mau also involve the respiratory muscles, leading to insufficient ventilation and admission to an intensive care unit. GBS pateints have a vairable prognosis; 20-30% require mechnical ventilation for a period ranging from weeks to months, 20% are unable to walk after 6 months nad 3-5% dies. Progression of weakness in GBS is usually rapid and reaches its peak within 4 weeks in the majority of patients, but many develop their maximum deficit within 2 weeks. Thereafter, the patients have a variable prognosis.
GBS is a treatable disorder. Intravenous immunoglobulin (IVIg) 2g/kg administered in 5 days was shown to be effective when administered within the first two weeks after onset of symptoms, and is considered the treatment of choice by most experts in the field. Although the standard treatment for GBS is a single course of IVIg (2g/kg administered in 5 days), many patients fails to recover abd remain with substantial disability. Patients with GBS and especially those with a poor prognosis potentially may benefit from more powerful abd when possible a more mechanistically rational therapy.
Recent experimental evidence suggests that complement activation palys a crucial role in the development of neuromuscular weakness in GBS making complement inhibitors and regulators attracive therapeutic targets. Our hypothesis is that Eculizumab, with its function as a complement inhibitor, will be very effective in preventing progression of weakness in patients with GBS.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Eculizumab | Experimental | Drug: EculizumabEculizumab, 900 mg intravenously once a week |
| Placebo | Placebo comparator | –Matched placebo, intravenously once a week |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Patients aged >18 years diagnosed with GBS according to NINDS diagnostic criteria
- Onset of weakness due to GBS is less than 2 weeks ago
- Patients who are unable to walk unaided for >10 metres (grade >3 on GBS disability scale)
- Patients who are being considered for or already on IVIg treatment
- First dose of eculizumab must be started within 2 weeks from onset of weakness and any time during the IVIg treatment period
Key exclusion criteria (10 listed)
- Age <18 years
- Patients who are being considered for, or already on, plasma exchange
- Pregnancy or lactation
- Patients show clear clinical evidence of a polyneuropahty caused by e.g. diabetes mellitus (except mild sensory), alcoholism, severe vitamin deficiency, and porphyria
- Patients received immunosuppressive treatment (e.g. azathioprine, cyclosporine, mycofenolatemofetil, tacrolimus, sirolimus or > 20 mg prednisolone daily) during the last month
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Eculizumab | Drug | Eculizumab |
Primary outcome
Determine the incidence of AE/SAEs after treatment with eculizumab and IVIg compared to placebo controls
Time frame: 6 months
Primary safety endpoint
Improvement of one or more grade in functional outcome (on the 6 point GBS disability scale) at 4 weeks
Time frame: 4 weeks
Primary efficacy endpoint
10 secondary outcomes
Ability to walk unaided (GBS disability score 2) at 8 weeks
Time frame: 8 weeks
Time taken to improve by at least one grade (on the GBS disability scale)
Time frame: 8 weeks
Time taken to walk independently
Time frame: 1 year
Difference in GBS disability score at maximum disability completed with 6 months
Time frame: 6 months
Percentage of patients with a clinically relevant improvement in R-ODS score
Time frame: 6 months
Percentage of patients with a clinically relevant improvement in ONLS
Time frame: 6 months
Requirement for ventilatory support (GBS disability score 5)
Time frame: 4 weeks
Duration of ventilatory support
Time frame: 8 weeks
Occurrence of relapse
Time frame: 2 years
Dearth within the first 6 months
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2014.
- Derived from this study: PubMed 27801990Davidson AI, Halstead SK, Goodfellow JA, Chavada G, Mallik A, Overell J, Lunn MP, McConnachie A, van Doorn P, Willison HJ. Inhibition of complement in Guillain-Barre syndrome: the ICA-GBS study. J Peripher Nerv Syst. 2017 Mar;22(1):4-12. doi: 10.1111/jns.12194.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Govindsinh Chavada contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eculizumab in people with Gullian Barre Syndrome, run by NHS Greater Glasgow and Clyde at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 23 Sep 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Govindsinh Chavada (govindsinh.chavada@glasgow.ac.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine the incidence of AE/SAEs after treatment with eculizumab and IVIg compared to placebo controls (time frame: 6 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02029378, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2014.