E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute Idiopathic Thrombocytopenic Purpura (ITP) in children |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10023095 |
E.1.2 | Term | ITP |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic 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 |
The primary objective is to investigate the hypothesis that early IVIG treatment in children with newly diagnosed acute ITP reduces the risk of development of chronic disease. |
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E.2.2 | Secondary objectives of the trial |
Secondary objectives are:
1. To evaluate the clinical parameters during the course of the disease, eg: bleeding score and time between onset of symptoms and recovery of platelet numbers.
2. Comparing the HRQoL in parents and patients with acute ITP who did and did not have IVIG and in those that do and do not develop chronic ITP.
3. Estimation of variability of biological parameters of the immune system of the patient that are supposed to be involved in the differences in outcome between acute vs. chronic disease as well as between response on IVIG treatment vs. non response. These include: A) the genetic polymorphisms of the activating and inhibiting IgG-Fc receptor and other inhibiting immune receptors. B) Immunoglobulin glycosylation variability within the platelet auto antibodies and its changes during time, as well as the changes due to IVIG treatment. C) Quantity and function of regulatory T cells.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
General inclusion criteria - Children aged 3 months -16 years, presenting to a pediatrician with newly diagnosed acute ITP and - Platelet count < 20 x 10 9 /L and - Bleeding tendency < grade 4 (Buchanan) and - no prior immunomodulating treatment within 4 weeks before diagnosis and - signed informed consent by parents and/ or patients
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E.4 | Principal exclusion criteria |
A patient presenting with any of the following criteria will not be included in the study:
General exclusion criteria - clinical features that are not compatible with the diagnosis of acute ITP, for example: presence of other auto-immune phenomena, organomegaly, other cytopenias besides thrombocytopenia or features susceptible for infectious disease like hepatitis, Epstein-Barr virus or HIV - immunomodulating treatment (IVIG, corticosteroids) within 4 weeks before diagnosis - history of allergic reactions against human plasma, plasma products or intravenous immunoglobulin - Severe or life threatening bleeding at presentation: grade 4 or 5 (Buchanan) - No informed consent
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary study endpoint is development of chronic ITP, defined by a platelet count of < 150 x 10^9/l six months after diagnosis. |
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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 | Yes |
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 | Yes |
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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
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 | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
careful observation without medication |
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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 | 90 |
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
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The trial ends after the last visit at 12 months after diagnosis. |
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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 | 5 |
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 |