|- candidate number||7789|
|- NTR Number||NTR2224|
|- ISRCTN||ISRCTN wordt niet meer aangevraagd.|
|- Date ISRCTN created|
|- date ISRCTN requested|
|- Date Registered NTR||24-feb-2010|
|- Secondary IDs||MEC-2009-368 METC Erasmus MC|
|- Public Title||Second IVIg Dose in Guillain-Barre syndrome patients with poor prognosis.|
|- Scientific Title||Second IVIg Dose in Guillain-Barre syndrome patients with poor prognosis.|
|- ACRONYM||SID-GBS trial|
|- hypothesis||To determine whether a second IVIg course in GBS patients with a poor prognosis improves functional outcome after 4 weeks. Secondary outcomes include functional outcome after 8, 12 and 26 weeks, mechanical ventilation, length of hospital and ICU admission, occurrence of TRF’s, mortality and blood IgG levels.|
|- Healt Condition(s) or Problem(s) studied||Guillain-Barre syndrome|
|- Inclusion criteria||To enter this GBS study:|
1. Patients are diagnosed with GBS;
2. There is an indication to start IVIg (irrespective of co-treatment with methylprednisolon (MP)) therapy:
A. Patient is unable to walk unaided for >10 meter (grade 3, 4 or 5 of the GBS disability scale), or;
B. There is otherwise an indication to start IVIg (with or without MP) treatment according to the treating neurologist.
3. Onset of weakness due to GBS is less than 2 weeks ago;
4. Signed informed consent.
To be randomized in the second IVIg dose phase (RCT), patients must fulfill the following criteria:
1. First IVIg (with or without MP) treatment with Nanogam® started within 2 weeks from onset of weakness;
2. IVIg treatment has been 2g/kg administered in 2-5 days;
3. Poor prognosis based upon the modified EGOS (mEGOS 6-12) at day 7 after start of first IVIg treatment.
|- Exclusion criteria||1. Age less than 6 years;|
2. Patient known to have a severe allergic reaction to properly matched blood products or plasma products;
3. Pregnancy or breastfeeding;
4. Patient known to have a selective IgA deficiency;
5. Patient shows clear clinical evidence of a polyneuropathy caused by e.g. diabetes mellitus (except mild sensory), alcoholism, severe vitamin deficiency, porphyria;
6. Patient received immunosuppressive treatment (e.g. azathioprine, cyclosporine, mycofenolaatmofetil, tacrolimus, sirolimus or > 20 mg prednisolon daily) during the last month;
7. Patient known to have a severe concurrent disease, like malignancy, severe cardiovascular disease, AIDS, severe CARA;
8. Inability to attend follow-up during 6 months.
|- mec approval received||yes|
|- multicenter trial||yes|
|- Type||2 or more arms, randomized|
|- planned startdate ||16-feb-2010|
|- planned closingdate||31-dec-2017|
|- Target number of participants||176|
|- Interventions||Second IVIg dose or placebo in selected patientgroup with a poor prognosis.|
|- Primary outcome||To determine whether a second IVIg dosage in GBS patients with a poor prognosis improve functional outcome after 4 weeks.|
|- Secondary outcome||To investigate whether: |
1. A second IVIg dosage in GBS patients with a poor prognosis improve functional outcome or muscle strength after 8, 12 and 26 weeks;
2. A second IVIg dosage in GBS patients with a poor prognosis lowers the percentage of patients needing artificial ventilation, lower the time (number of days) on respirator or time on the intensive care;
3. A second IVIg dosage in GBS patients with a poor prognosis reduces the time to hospital discharge;
4. A second IVIg dosage in GBS patients with a poor prognosis reduce the chance of secondary deterioration due to treatment-related fluctuations (TRF†);
5. Patients treated with a second IVIg dosage develop more complications possibly related to the second IVIg treatment;
6. A second IVIg dosage in GBS patients with a poor prognosis lowers the percentage of patients that die because of GBS;
7. The serum IgG increase after the first IVIg dosage is lower in patients with a poor prognosis;
8. Serum IgG increases further (and to what extent) after administration of a second IVIg dosage.
|- Timepoints||Admission and 1, 2, 4, 8, 12 and 26 weeks after admission.|
|- Trial web site||N/A|
|- status||open: patient inclusion|
|- CONTACT FOR PUBLIC QUERIES|| Christa Walgaard|
|- CONTACT for SCIENTIFIC QUERIES|| P.A. Doorn, van |
|- Sponsor/Initiator ||Erasmus Medical Center, Rotterdam|
(Source(s) of Monetary or Material Support)
|Prinses Beatrix Fonds, Sanquin Blood Supply|
|- Brief summary||Rationale:|
Guillain-Barré syndrome (GBS) is the most frequent cause of acute neuromuscular weakness in the Western world. GBS patients have a variable prognosis, 20-30% needs mechanical ventilation, 20% is unable to walk after 6 months and 3% dies. Using a simple scoring system it is
possible to accurately predict which patient has a poor prognosis. GBS patients with a poor prognosis may benefit from a second course of IVIg.
To determine whether a second IVIg course in GBS patients
with a poor prognosis improves functional outcome after 4 weeks.
Secondary outcomes include functional outcome after 8, 12 and 26 weeks, mechanical ventilation, length of hospital and ICU admission, occurrence of TRF’s, mortality and blood IgG levels.
A double-blind randomized placebo-controlled trial
design will be used in selected patients with a poor prognosis. In patients with a good prognosis the study will have an observational design.
GBS patients of 6 years and older, who have an
indication for IVIg treatment.
Patients with a poor prognosis according to the
prediction model (mEGOS) will be randomized to receive a second IVIg course or placebo.
Main study parameters/endpoints:
The main study endpoint is functional outcome on the GBS disability scale 4 weeks after start of the first IVIg course. Other endpoints include functional outcome after 26 weeks.
Nature and extent of the burden and risks associated with
participation, benefit and group relatedness:
The participant will be physically examined at 7 standardized time-points, mostly in combination
with the standard patient care. Extra blood and CSF (extra spinal tab is not requested) will be used for trial purposes. An EMG will be performed in the standard clinical work-up. A throat swab will be requested. Serious adverse effects of IVIg are rare (details in SPC). It is anticipated that also in children benefits will outweigh the risks of participating in this treatment trial. Standard treatment in children is
the same as in adults, long-term prognosis is better as in adults; however 25% is still not symptom free at a median of 228 days in a large observational study.
|- Main changes (audit trail)|
|- RECORD||24-feb-2010 - 5-jun-2016|