E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) |
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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 | 10028533 |
E.1.2 | Term | Myelodysplastic syndrome |
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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 is to provide long-term safety data for the use of romiplostim in thrombocytopenic subjects with myelodysplastic syndromes (MDS). |
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E.2.2 | Secondary objectives of the trial |
The secondary objective is to evaluate the effectiveness of romiplostim with respect to platelet response, transfusion, and bleeding events in the treatment of thrombocytopenic subjects with MDS. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Disease related: • Subject completed a romiplostim study for the treatment of thrombocytopenia in subjects with MDS Demographic: • Eastern Cooperative Oncology ( ECOG) performance status of 0-2 Laboratory: • Subject had a platelet count of ≤ 50 x 109/L since the final dose of investigational product in the parent study General: • Subject or his/her legally acceptable representative provided written informed before any study-specific procedures were initiated |
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E.4 | Principal exclusion criteria |
Disease Related: • Subject has been diagnosed with AML or has a blast count ≥ 20% by peripheral blood or bone marrow biopsy • Subject has prior history of leukemia • Prior history of bone marrow or stem cell transplantation • Prior malignancy (other than in situ cervical cancer, controlled prostate cancer, or basal cell cancer of the skin) unless treated with curative intent and without evidence of disease for ≥ 3 years before randomization • Active or uncontrolled infections • Unstable angina, congestive heart failure [NYHA > class II], uncontrolled hypertension [diastolic > 100 mmHg], uncontrolled cardiac arrhythmia, or recent (within 1 year) myocardial infarction • History of arterial thrombosis (eg, stroke or transient ischemic attack) in the past year • History of venous thrombosis that currently requires anti-coagulation therapy Medications: • Received IL-11 within 4 weeks of screening • Subject previously received a thrombopoietic growth factor (other than romiplostim) • Known hypersensitivity to any recombinant E coli-derived product (eg, Infergen, Neupogen, Somatropin, and Actimmune) General: • Subject currently is enrolled in or has not yet completed at least four weeks since ending investigational device or drug study(s) (other than romiplostim), or subject is receiving other investigational agent(s) • Subject of child-bearing potential is evidently pregnant (eg, positive HCG test) or is breast feeding • Subject is not using adequate contraceptive precautions • Subject has any kind of disorder that compromises his/her ability to give written informed consent (and does not have a legally acceptable representative; see Section 12.1) or is unable to comply with study procedures |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the incidence of all 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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 6 |
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 | 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 |