E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Adult immune thrombocytopenic purpura (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 |
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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 describe the number of months with a subject platelet response over a 12 month treatment period |
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E.2.2 | Secondary objectives of the trial |
• To describe the subject incidence of ITP remission • To describe the subject incidence of splenectomy • To describe the subject incidence of adverse events |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Subject has been diagnosed with primary ITP according to the ASH guidelines (George et al, 1996) and previously received only 1st line therapies First line therapy is defined as corticosteroids, IVIG, Anti-D and Vinca Alkaloids (used for the treatment of ITP related thrombocytopenia only). A platelet transfusion at any time during the six month period since the original diagnosis would not exclude the subject from study participation 4.1.2 Initial diagnosis of primary ITP within 6 months of enrollment 4.1.3 Age ≥ 18 years at screening 4.1.4 A single platelet count ≤ 30 x 109/L at any time during the screening period 4.1.5 Subject or subject’s legally acceptable representative has provided informed consent |
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E.4 | Principal exclusion criteria |
Known history of a bone marrow stem cell disorder ������ Any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study 4.2.2 Surgical resection of the spleen 4.2.3 Subject has a history of cancer or current malignancy other than basal cell carcinoma or cervical cancer in-situ with active treatment or disease within 5 years of screening 4.2.4 Known history of congenital thrombocytopenia 4.2.5 Known history of hepatitis B, hepatitis C, or human immunodeficiency virus 4.2.6 Positive H. pylori by urea breath test or stool antigen test at screening 4.2.7 Known history of systemic lupus erythematosus, Evans syndrome, or autoimmune neutropenia 4.2.8 Known history of antiphospholipid antibody syndrome or positive for lupus anticoagulant 4.2.9 Known history of disseminated intravascular coagulation, hemolytic uremic syndrome, or thrombotic thrombocytopenic purpura 4.2.10 Previous history of recurrent venous thromboembolism or thrombotic events or an occurrence within 5 years of enrollment. 4.2.11 Previous use of romiplostim, PEG-rHuMGDF, eltrombopag, rHuTPO or any platelet producing agent 4.2.12 Rituximab (for any indication) or Mercaptopurine (6-MP) or anticipated use during the time of the proposed study 4.2.13 All hematopoietic growth factors including IL-11 (oprelvekin) within 4 weeks before the screening visit 4.2.14 Alkylating agents use at any time before or during the screening visit or anticipated during the time of the proposed study 4.2.15 Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune) 4.2.16 Currently enrolled in another investigational device or drug study, or less than 30 days since ending another investigational device or drug study(s), or receiving other investigational agent(s) 4.2.17 Subject will have any other investigational procedures performed while enrolled in this clinical study 4.2.18 Subject is pregnant or breast feeding, or planning to become pregnant within 5 weeks after the end of treatment 4.2.19 Female subject of child bearing potential is not willing to use, in combination with her partner, highly effective contraception during treatment and for 5 months after the end of treatment 4.2.20 Subject has previously enrolled into a romiplostim study 4.2.21 Subject will not be available for protocol-required study visits, to the best of the subject’s and investigator’s knowledge 4.2.22 Subject has any kind of disorder that, in the opinion of the investigator, may compromise the ability of the subject to give written informed consent and/or to comply with all required study procedures |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the number of months with platelet response during the 12 month treatment period. A platelet response during any one month is defined as the median of platelet counts measured in the month ≥ 50 x 109/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 | 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 | 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 | 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 30 |
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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La conclusione della sperimentazione coincide con l`ultima visita dell`ultimo paziente (LPLV - Last Patient Last Visit). |
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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 | 6 |
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 | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |