E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Immune thrombocytopenic purpura (ITP) is the most common autoimmune disease. It is a bleeding disorder characterized by decreased counts of circulating platelets and normal or increased numbers of megakaryocytes in the bone marrow. |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The study is designed to assess the efficacy, tolerability and safety of IgPro10 |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
Male or female patients aged 18 – 65 years
Diagnosis of chronic ITP defined by:
Failure to find other causes of thrombocytopenia
Platelet count ≤ 150 x 10 (9) /L over 6 months or response to a previous treatment with subsequent decrease in platelet count even if duration of chronic ITP is less than 6 months
Platelet counts ≤ 20 x 10 (9) /L
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E.4 | Principal exclusion criteria |
Treatment with IVIG or anti-D immunoglobulin within 3 weeks prior to screening.
Treatment with IV steroids within 10 days prior to screening.
Change of oral steroid treatment within 15 days prior to screening.
Patients with known or suspected hypersensitivity to immunoglobulins or previous severe side effects to immunoglobulin therapy.
Patients with a history of migraine.
Patients with known hyperprolinemia.
Abnormal results in the following laboratory parameters: Hemoglobin < 10 g/dL Total bilirubin > 1.5 x upper normal limit ALAT > 2.5 x upper normal limit ASAT > 2.5 x upper normal limit Creatinine > 1.5 x upper normal limit Urea > 1.5 x upper normal limit
Patients with low serum IgA level, defined by < 50% lower normal limit.
Patients with one of the following concomitant diseases: Clinical active SLE Lymphoproliterative disease Heart failure Grade III or IV according to the New York Heart Association classification
Any other concomitant disease that has influence on the clotting system (i.e. hemophilia) |
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E.5 End points |
E.5.1 | Primary end point(s) |
Response defined by an elevation of platelet count within 7 days after the first study drug administration to at least 50 x 10 (9) /L |
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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 | No |
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 | Information not present in EudraCT |
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 | Yes |
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 | No |
E.8.1.1 | Randomised | No |
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 | 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 | No |
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.8.5 | The trial involves multiple Member States | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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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It is foreseen in the protocol that patients are followed for four weeks after the administration of the study drug. For all the patients having a platelet count of ≥ 50 x 10 (9) /L at Day 29 the follow-up period will be prolonged for additional four (Day 57) to eight weeks (Day 85), or until the platelets fall below 50 x 10 (9) /L. In addition to the platelet count measurements, bleeding events, concomitant medication and adverse events will also be documented at those visits. |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 0 |