Clinical trial NCT00117143: Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
Official title: An Open-label, Unit Dose-finding Study Evaluating the Safety and Efficacy of Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Thrombocytopenic Purpura
Keywords: Immune Thrombocytopenic Purpura
Idiopathic Thrombocytopenic Purpura
ITP
Thrombocytopenia
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2005-07-04
Enrollment: 16
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the safety and tolerability of AMG 531 (romiplostim), a novel thrombopoiesis-stimulating peptibody, and its effect on platelet counts in adults with immune thrombocytopenic purpura.


Eligibility criteria: Inclusion Criteria:

* Greater than or equal to 3 months history of ITP, regardless of splenectomy status, and completion of at least 1 prior treatment for ITP
* 2 of 3 pretreatment platelet counts that were less than 30 x 10\^9/L (if not currently on ITP therapy) or less than 50 x 10\^9/L (if currently receiving corticosteroids for ITP therapy)
* Ability to give informed consent

Exclusion Criteria:

* Known history of arterial thrombosis, active malignancy, or bone marrow stem cell disorder

Primary outcomes:
measure: Number of Participants With Adverse Events
timeFrame: From first dose through 8 weeks after last dose of study drug (11 weeks)
measure: Number of Participants With Positive Anti-Romiplostim Antibodies
timeFrame: Days 29 and 78
Secondary outcomes:
measure: Number of Participants Who Achieved a Targeted Therapeutic Platelet Response
timeFrame: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
measure: Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L From Baseline
timeFrame: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
measure: Number of Participants With Peak Platelet Counts of ≥ 100 x 10⁹ Cells/L
timeFrame: After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants)
measure: Number of Participants With Peak Platelet Counts of ≥ 450 x 10⁹ Cells/L
timeFrame: After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants)
measure: Change From Baseline to Peak Platelet Level
timeFrame: Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants)
measure: Time to Peak Platelet Count
timeFrame: From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78
measure: Duration Within the Targeted Therapeutic Range
timeFrame: From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78
Other outcomes:
Locations:
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: 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.
citation: Newland A, Caulier MT, Kappers-Klunne M, Schipperus MR, Lefrere F, Zwaginga JJ, Christal J, Chen CF, Nichol JL. An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. Br J Haematol. 2006 Nov;135(4):547-53. doi: 10.1111/j.1365-2141.2006.06339.x.