E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Thrombocytopenia in subjects with Immune Thrombocytopenic Purpura (ITP) |
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MedDRA Classification |
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 evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by durable platelet response during the last 8 weeks of treatment and other platelet response parameters. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the overall safety of AMG 531
To evaluate possible reductions in the dose of concurrent ITP therapies while receiving AMG 531
To evaluate changes in Patient Reported Outcomes (PRO) and Health Resource Utilization (HRU) due to treatment with AMG 531 |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1.·Diagnosis of ITP according to American Society of Hematology (ASH) guidelines at least 3 months before enrollment 2. Have completed at least 1 prior treatment for ITP (eg, prednisone) 3. Subjects > 60 years of age must have a minimum of one year documented history of chronic ITP with a bone marrow report to confirm the diagnosis 4. The mean of the three platelet counts taken during the screening and pre-treatment periods must be ≤ 30 x 109/L, with no individual count > 35 x 109/L 5. Subjects must be greater than or equal to 18 years of age at the time of obtaining the informed consent 6. A serum creatinine concentration less than or equal to 2 mg/dl (less than or equal to 176.8 mmol/L) 7. Adequate liver function, as evidenced by a serum bilirubin less than or equal to 1.5 times the laboratory normal range 8. Hemoglobin ≥ 9.0 g/dL 9. Before any study-specific procedure, the appropriate written informed consent must be obtained
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E.4 | Principal exclusion criteria |
1. Have had a Splenectomy for any reason 2. Any known history of 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) 3. Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years before randomization 4. Currently receiving any treatment for ITP, except corticosteroids, azathioprine, or danazol administered at a constant dose and schedule 5. IV Ig or anti-D Ig within 2 weeks before the screening visit 6. Rituximab (for any indication) within 14 weeks before the screening visit or anticipated use during the time of the proposed study 7. Received hematopoietic growth factors, including IL-11 (oprelvekin) within 4 weeks before the screening visit 8. Received any alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study 9. Subject is currently enrolled in or has not yet completed at least 4 weeks since ending other investigational device or drug trial(s), or subject is receiving investigational agent(s) other than AMG 531 10. Past or present participation in any study evaluating PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO), AMG 531, or related platelet product 11. Subject of child-bearing potential is evidently pregnant (eg, positive HCG test) or is breast feeding 12. Subject is not using adequate contraceptive precautions 13. Known hypersensitivity to any recombinant E coli -derived product 14. Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and/or to comply with all study procedures
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the incidence of durable platelet response. A subject with durable platelet response is defined as achieving at least 6 weekly platelet responses during the last 8 weeks of treatment in the absence of rescue medication. A weekly platelet response is defined as a platelet count of ≥ 50 x 109/L on the weekly scheduled dose day from week 2 to week 25.
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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 | No |
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 | Information not present in EudraCT |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
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.8.5 | The trial involves multiple Member States | Yes |
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 | Information not present in EudraCT |
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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All subjects completing the 24 week treatment period will have their platelet counts monitored starting at week 26. Subjects whos platelet count has not dropped to less than or equal to 50 x 109/L by week 26 will return to the clinic at scheduled weeks 27, 28, 31 and 36. Subjects will complete the study when platelet counts have dropped to less than or equal to 50 x 109/L, or at week 36, which ever is reached first. |
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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 | |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 11 |