E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Low or Intermediate 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 |
To provide long-term safety data for the use of romiplostim in thrombocytopenic subjects with IPSS low or intermediate-1 risk 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 IPSS low or intermediate-1 risk MDS. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Disease related: • Completion of a romiplostim study including the End of Study visit for the treatment of thrombocytopenia in subjects with IPSS low to int-1 risk MDS (e.g. 20050159 or 20060198) Demographic: • Eastern Cooperative Oncology ( ECOG) performance status of 0-2 Laboratory: • Adequate Liver Function, as evidenced by a serum bilirubin ≤ 2 times the laboratory normal range and unconjugated bilirubin ≥90% of total bilirubin (except for patients with a confirmed diagnosis of Gilbert’s Disease), ALT ≤ 3 times the laboratory normal range, and AST ≤ 3 times the laboratory normal range • A serum creatinine concentration ≤ 2 mg/dl • Platelet count ≤ 50 x 109/ L General: • Written informed consent |
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E.4 | Principal exclusion criteria |
Disease Related: • Evidence of progression/transformation of disease (i.e. increase in bone marrow myeloblasts by ≥ 5% or worsening cytogenetics since screening for parent study) • Prior history of leukemia or aplastic anemia • 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 • Receipt of hypomethylating agents or immunomodulating agents, high-dose chemotherapy targeted at MDS, or histone deacetylase inhibitors • Have previously received any other thrombopoietic growth factor • 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 4 weeks since ending investigational device or drug study(s) (other than AMG 531), 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 the ability of the subject to give written informed consent and/or 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 AEs, 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 | 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 | Yes |
E.7.1.3.1 | Other trial type description |
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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 | 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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 7 |
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
| |
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 | 0 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |