E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
AutoImmune Hemolytic Anemia |
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E.1.1.1 | Medical condition in easily understood language |
AutoImmune Hemolytic Anemia |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10003825 |
E.1.2 | Term | Autoimmune hemolytic anemia |
E.1.2 | System Organ Class | 100000004851 |
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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 |
to test whether co-administration of C1-inh concentrate in patients suffering from AIHA needing RBC transfusion • improves recovery of RBC transfusion • inhibits complement activation and deposition on RBC via the classical pathway of complement • is safe?
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E.2.2 | Secondary objectives of the trial |
to investigate whether co-administration of C1-inhibitor concentrate in patients suffering from AIHA needing RBC transfusion • attenuates the pro-inflammatory response in AIHA • affects the response to the basic treatment targeting autoantibody production
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Positive (≥1+) monospecific antiglobulin test for C3b and/or C3d with/without positivity for IgM or IgG • Indication for a transfusion with at least 2 red packed cell concentrates based on the clinical assessment by the hematologist in charge • Hemoglobin value at least < 5 mmol/L (8 g/dl) with/without clinical symptoms • Clinical signs of hemolysis: not-detectable haptoglobin (mandatory) and increased lactate dehydrogenase (LDH) eventually combined with hyperbilirubinemia (increased direct and/or indirect bilirubin), lactate. • Age ≥ 18 years • Written informed consent • Women of child bearing potential must have had a negative serum pregnancy test 7 days prior to the start of study drug
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E.4 | Principal exclusion criteria |
• History of arterial and/or venous thromboembolic events in the absence of an actual treatment with Vitamin K-antagonists • Concomitant use of therapeutic doses of heparin • Female patients who are pregnant or breast feeding or adults of reproductive potential who are not using effective birth control methods. If barrier contraceptives are being used, these must be continued throughout the trial by both sexes. Oral contraceptives only are not acceptable. • Patients with known HIV seropositivity or chronic active hepatitis • Patients who have any severe and/or uncontrolled medical condition or other conditions that could affect their participation in the study such as: – cerebrovascular accidents ≤ 6 months before study drug start – uncontrolled hypertension
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E.5 End points |
E.5.1 | Primary end point(s) |
• improves recovery of RBC transfusion • inhibits complement activation and deposition on RBC via the classical pathway of complement
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
• At entry: laboratory tests within 4 hours before start of treatment, before and after every dose C1-inh as well as 48 hours, 60 hours and 72 hours after the first dose NB: in case that the time between study entry and start treatment exceeds 4 hours, laboratory tests performed at study entry must be repeated
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | 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 | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
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) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 | 2 |
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 | No |
E.8.7 | Trial has a data monitoring committee | No |
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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
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 | 2 |
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 |