E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Immune (Idiopathic) Thrombocytopenic Purpura |
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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 |
The primary objective of this study is to evaluate the safety of AMG 531 (Romiplostim) in severely refractory trombocytopenic subjects with ITP. |
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E.2.2 | Secondary objectives of the trial |
This study will also evaluate hematological responses to Romiplostim. In addition, the study will provide open label use of Romiplostim, and will investigate its utility in severely refractory thrombocytopenic subjects with immune (idiopathic) thrombocytopenic purpura (ITP) who do not qualify for ongoing ITP studies using Romiplostim. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion Criteria • Subject is ≥ 18 years of age • Subject has a diagnosis of immune (idiopathic) thrombocytopenic purpura per the American Society of Hematology guidelines • Subject had a bone marrow biopsy and aspirate consistent with ITP conducted within 2 years of Screening • Subject’s platelet count is ≤ 20 x10^9/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, is unlikely 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 |
Exclusion Criteria • Subject has a history of arterial thrombosis (eg, cerebrovascular accident, transient ischemic attack, or 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 PEG-rHuMGDF, 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 subject’s ability to comply with the protocol procedures |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the incidence of adverse events, including clinically significant changes in laboratory values and incidence of antibody formation. |
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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 | 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 | 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.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 100 |
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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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 | 5 |