E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acquired Thrombotic Thrombocytopenic Purpura (TTP). This population includes symptomatic patients with acute episodes of idiopathic TTP as well as secondary TTP syndrome in association with clinical conditions or medicinal products (de novo and recurrent symptoms) requiring treatment with plasma exchange. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10043648 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10043648 |
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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 |
Reduction of time-to-recovery, defined by the achievement of laboratory blood marker response, confirmed at 48 hours after the initial reporting of this response |
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E.2.2 | Secondary objectives of the trial |
Improvement in number of patients responding to therapy � Reduction in, plasma exchange (PE) procedure- related items � Reduction of time to resolution or improvement of symptoms typical of TTP, including blood markers � Reduction of number of exacerbations and relapses � Improvement of cognitive level at steady state post acute phase � Improvement of clinical symptoms and organ function � Reduction in mortality at 30 days following PE. � Reduction of concomitant treatment-related complications � Evaluation of safety and immunogenicity of adjunctive treatment with ALX-0081 Determination of pharmacokinetic (PK) and pharmacodynamic (PD) characteristics of ALX-0081 in patients with acquired TTP |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. 18 years of age or older 2. Men or women willing to accept an acceptable contraceptive regimen 3. Patients with clinical diagnosis of TTP 4. Necessitating plasma exchange 5. Patient accessible to FU 6. Obtained, signed and dated informed consent |
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E.4 | Principal exclusion criteria |
1. Platelet count greater or equal to 100,000/μL 2. Severe active infection indicated by sepsis (requirement for pressors with or without postive blood cultures) 3. Clinical evidence of enteric infection with E.coli 0157 or related organism 4. Anti-phospolipid syndrome 5. Diagnosis of disseminated intravascular coagulation (DIC) 6. Pregnancy or breast-feeding 7. Haematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy 8. Known congenital TTP 9. Active bleeding or high risk of bleeding 10. Uncontrolled arterial hypertension 11. Known chronic treatment with anticoagulant treatment that can not be stopped safely, including but not limited to: � vitamin K antagonists � heparin or LMWH � non-acetyl salicylic acid non-steroidal anti-inflammatory molecules 12. Severe or life threatening clinical condition other than TTP that would impair participation in the trial 13. Subjects with malignancies resulting in a life expectation of less than 3 months 14. Subjects with bone marrow carcinosis 15. Subjects who cannot comply with study protocol requirements and procedures. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Time to response, based on the following criteria: - Recovery of platelets ≥ 150,000/μL - This response must be confirmed at 48 hours after the initial reporting of platelet recovery above 150,000/μL by a de novo measure of platelets ≥ 150,000/μL and LDH ≤ 2 X ULN |
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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 | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
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 | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | Yes |
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 | 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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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 | 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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ULTIMA VISITA dell`ULTIMO PAZIENTE |
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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 | 9 |
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 | 9 |
E.8.9.2 | In all countries concerned by the trial days | 0 |