E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Thrombocytopenia in subjects with ITP |
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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 | 10021245 |
E.1.2 | Term | Idiopathic thrombocytopenic purpura |
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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 demonstrate the safety of AMG 531 in severely refractory thrombocytopenic subjects with ITP |
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E.2.2 | Secondary objectives of the trial |
To monitor hematological responses to AMG 531 To provide open-label use of AMG 531, and to investigate its utility in severely refractory thrombocytopenic subjects with ITP who do not qualify for any ongoing ITP studies with AMG 531 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subject is ≥ 18 years of age Subject has a diagnosis of immune (idiopathic) thrombocytopenic purpura per the American Society of Hematology guidelines (see Appendix B) Subject had a bone marrow biopsy and aspirate consistent with ITP conducted within 2 years of Screening Subjects platelet count is ≤ 20 x109/L or the subject is experiencing bleeding that is uncontrolled with conventional therapies Subject has failed at least 3 conventional therapies for ITP and, in the opinion of the treating physician, isunlikely to respond to other available therapies If a subject has a history of atrial fibrillation, subject is currently receiving anti-coagulation medication Subject (or legally-acceptable representative) is willing and able to provide written informed consent |
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E.4 | Principal exclusion criteria |
Subject has a history of arterial thrombosis (eg, cerebrovascular accident, transient ischemic attack, myocardial infarction) Subject has a history of venous thrombosis (eg, deep vein thrombosis, pulmonary embolism) Subject has a history of human immunodeficiency virus, hepatitis B virus, hepatitis C virus, or any other systemic infectious disease known to cause severe thrombocytopenia Subject has a history of disseminated intravascular coagulation or underlying hypercoaguable state Subject has a history of any of the following autoimmune disorders: systemic lupus erythematosis, Evans Syndrome, autoimmune neutropenia, lupus anticoagulant or antiphospholipid antibody syndrome, or active vasculitis Subject has a history of microangiopathic hemolytic anemia (ie, hemolytic uremic syndrome, thrombotic thrombocytopenic purpura) Subject has active lymphoproliferative or immunoproliferative (monoclonal gammopathy of undeterimined significance, multiple myeloma) disorder or leukemia Subject has a history of a myeloproliferative disorder (eg, myelofibrosis, chronic myelogenous leukemia) Subject has myelodysplastic syndrome Subject with a history of exposure to mutagenic chemotherapy has either dysplastic cytological findings or abnormal cytogenetics on bone marrow study Subject has a history of paroxysmal nocturnal hemoglobinuria Subject has participated in any study evaluating PEGrHuMGDF, recombinant human thrombopoietin (rHuTPO), or related platelet product Subject has a known hypersensitivity to any recombinant E coli-derived product Subject has received any therapeutic drug or device that is not approved by the local regulatory health agency for any indication within 4 weeks of Screening Subject is of reproductive potential and is not using adequate contraceptive precautions, in the judgment of the investigator Subject is pregnant or breast feeding Investigator has concerns regarding the subjects ability to comply with the protocol procedures |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Objective To demonstrate the safety of AMG 531 in severely refractory thrombocytopenic subjects with ITP |
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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 | 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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 23 |
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 | 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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Fino a quando il farmaco sara' disponibile in commercio |
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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 | 6 |
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 | 6 |