E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Guillian-Barre syndrome |
Guillain-Barré Syndroom |
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E.1.1.1 | Medical condition in easily understood language |
Auto-immune demyelinating polyneuropathy |
auto-immuun gemedieerde demyeliniserende polyneuropathie |
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E.1.1.2 | Therapeutic area | Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutic techniques [E02] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10018767 |
E.1.2 | Term | Guillain-Barre syndrome |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine whether a second IVIg dosage in GBS patients with a poor prognosis improve functional outcome after 4 weeks. |
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E.2.2 | Secondary objectives of the trial |
-functional outcome or muscle strength after 8, 12 and 26 weeks. -the percentage of patients needing artificial ventilation, lower the time (number of days) on respirator or time on the intensive care. -reduces the time to hospital discharge. -reduce the chance of secondary deterioration due to treatment-related fluctuations (TRF†). -development of more complications possibly related to the second IVIg treatment. -lowers the percentage of patients that die because of GBS. -the serum IgG increase after the first IVIg dosage is lower in patients with a poor prognosis. -serum IgG increases further (and to what extent) after administration of a second IVIg dosage.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
diagnosed with GBS according to the international accepted criteria, given informed consent, patient in grade 3, 4 or 5 of the GBS disability scale or is there otherwise an indication for IVIg therapy according to the treating neurologist, less than two weeks since onset of weakness, able to attend follow-up of 6 months. |
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E.4 | Principal exclusion criteria |
age below 6 years, severe allergic reaction to properly matched blood products, known to have severe concurrent disease, having selective IgA deficiency, clinical evidence of polyneuropathy caused by other cause than GBS, received immune suppressive therapy during last month, pregnant or breast feeding. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Main study endpoint: GBS disability score at 4 weeks after start of first IVIg course. The full range of scores will be considered as an ordinal outcome scale. In analyzing we will use a proportional odds model. (Extent of) improvement on this ordinal scale will be compared between groups.
Secondary study endpoints: -Percentage of patients that improve: at least 1, 2, 3 or 4 points on the GBS disability score at 4, 8, 12 and 26 weeks, at least 4, 8 or 12 points on MRC sum score (ranging from 0-60) at 4, 8, 12 and 26 weeks, at least 2, 4 or 6 points on ONLS score (ranging from 0-12) at 4, 8, 12 and 26 weeks. -Percentage of patients needing artificial ventilation. -Time (number of days) on respirator. -Time (number of days) on intensive care unit. -Percentage of patients that die because of GBS. -Time (number of days) to hospital discharge. -Percentage of patients with secondary deterioration due to treatment-related fluctuations (TRF). -Development of complications possibly related to a second IVIg course. -Serum IgG levels at 5 different time points. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 70 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
When 44 subjects have received a second dosage IVIg the trial will be stopped. |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |