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 |
To provide long-term safety data for the use of AMG 531 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 AMG 531 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 an End of Study visit in AMG 531 study 20050159 or 20060198 for the treatment of thrombocytopenia in subjects with IPSS low to int-1 MDS. Demographic: • Eastern Cooperative Oncology ( ECOG) performance status of 0-2 Laboratory: • Adequate Liver Function, as evidenced by a serum bilirubin ≤ 1.5 times the laboratory normal range ( except for patients with a confirmed diagnosis of Gilbert’s Disease) or an ALT and AST ≤ 3 times the laboratory normal range • A serum creatinine concentration ≤ 2 mg/dl • Platelet count ≤ 50 x 10-9/ L General: • Written informed consent |
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E.4 | Principal exclusion criteria |
Disease Related: • Evidence of progression/transformation of disease (ie increase in bone marrow myeloblasts by ≥ 5% or worsening cytogenetics) • 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 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 | 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 |