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The purpose of this study is to evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by the platelet response. This study will also evaluate changes in Patient Reported Outcomes and Health Resource Utilization due to treatment with AMG 531.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| AMG 531 | Experimental | Active investigational product |
|
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AMG 531 | Biological | Weekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. AMG 531 is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Last 8 weeks of treatments |
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the overall safety of AMG 531 | Entire duration of the study including the follow up period | |
| To evaluate possible reductions in the dose of concurrent ITP therapies while receiving AMG 531 | Entire duration of the study |
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Inclusion Criteria:
Diagnosis of ITP according to American Society of Hematology (ASH) guidelines (Appendix F)
Have completed as least 1 prior treatment for ITP (e.g., prednisone)
Subjects greater than 60 years of age must have a documented history of chronic ITP with a bone marrow report to confirm the diagnosis
The platelet count (calculated from the mean of the 2 counts taken during the screening and pre-treatment periods) must be:
*less than 30 x 10^9/L for those subjects not receiving any ITP therapy, with no count greater than 35 x 10^9/L *less than 50 x 10^9/L for those subjects receiving a constant dose schedule of corticosteroids, azathioprine or danazol with no count greater than 55 x 10^9/L
A serum creatinine concentration less than or equal to 2 mg/dl (less than or equal to 176.8 µmol/L)
Adequate liver function, as evidenced by a serum bilirubin less than or equal to 1.5 times the laboratory normal range
Hemoglobin greater than 11.0 g/dL
Written informed consent (see Section 12.1)
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| MD | Amgen | Study Director |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28411254 | Background | Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, Lyons R, Garcia-Chavez J, Carpenter N, Wang X, Eisen M. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017 Aug;102(8):1342-1351. doi: 10.3324/haematol.2016.161968. Epub 2017 Apr 14. | |
| 30793285 |
| Label | URL |
|---|---|
| AmgenTrials clinical trials website | View source |
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| ID | Term |
|---|---|
| D013921 | Thrombocytopenia |
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| D011696 | Purpura, Thrombocytopenic |
| ID | Term |
|---|---|
| D001791 | Blood Platelet Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000095542 | Cytopenia |
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| ID | Term |
|---|---|
| C488777 | romiplostim |
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| Placebo | Drug | Weekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. Placebo is supplied as a lyophilized power in a 5 mL single use glass vial. |
|
| To evaluate changes in Patient Reported Outcomes (PRO) and Health Resource Utilization (HRU) due to treatment with AMG 531 | Entire duration of the study |
| Kuter DJ, Newland A, Chong BH, Rodeghiero F, Romero MT, Pabinger I, Chen Y, Wang K, Mehta B, Eisen M. Romiplostim in adult patients with newly diagnosed or persistent immune thrombocytopenia (ITP) for up to 1 year and in those with chronic ITP for more than 1 year: a subgroup analysis of integrated data from completed romiplostim studies. Br J Haematol. 2019 May;185(3):503-513. doi: 10.1111/bjh.15803. Epub 2019 Feb 21. |
| 32129511 | Background | Kuter DJ, Arnold DM, Rodeghiero F, Janssens A, Selleslag D, Bird R, Newland A, Mayer J, Wang K, Olie R. Safety and efficacy of self-administered romiplostim in patients with immune thrombocytopenia: Results of an integrated database of five clinical trials. Am J Hematol. 2020 Jun;95(6):643-651. doi: 10.1002/ajh.25776. Epub 2020 Mar 21. |
| 22494019 | Derived | Weitz I, Sanz MA, Henry D, Schipperus M, Godeau B, Northridge K, Gleeson M, Danese M, Deuson R. A novel approach to the evaluation of bleeding-related episodes in patients with chronic immune thrombocytopenia. Curr Med Res Opin. 2012 May;28(5):789-96. doi: 10.1185/03007995.2012.684046. Epub 2012 Apr 25. |
| 19671919 | Derived | Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009 Oct 29;114(18):3748-56. doi: 10.1182/blood-2009-05-224766. Epub 2009 Aug 11. |
| 18242413 | Derived | Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrere F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. doi: 10.1016/S0140-6736(08)60203-2. |
| FDA-approved Drug Labeling | View source |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D057049 | Thrombotic Microangiopathies |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |