Amgen Megakaryopoiesis Protein 2 (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)

Thrombocytopenic Purpura Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00117143
• Other study ID #: 20010218
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: December 2, 2002
• Est. completion date: July 19, 2004

Study information

• Verified date: November 2019
• Source: Amgen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: July 19, 2004
• Est. primary completion date: July 19, 2004
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Purpura
• Purpura, Thrombocytopenic
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenic Purpura

Intervention

Drug:
• Romiplostim
Administered subcutaneously on day 1 and on day 15 or 22 if the platelet count was = 50 x 10?/L and not rising, peak platelet count was = 450 x 10?/L and no serious adverse events related to treatment were observed.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Amgen

References & Publications (2)

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. — View 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events		From first dose through 8 weeks after last dose of study drug (11 weeks)
Primary	Number of Participants With Positive Anti-Romiplostim Antibodies	The presence or development of antibodies to romiplostim and endogenous thrombopoietin was assessed using a neutralizing bioassay. Antibody analyses were conducted on study days 29 and at the end-of-study visit (day 78). The number of participants with positive antibody binding at any time during the study is reported.	Days 29 and 78
Secondary	Number of Participants Who Achieved a Targeted Therapeutic Platelet Response	Targeted therapeutic platelet response was defined as a (single) platelet count that was double the baseline level and between 50 and 450 × 10? cells/L.; Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.	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)
Secondary	Number of Participants With an Increase in Platelet Count of = 20 x 10? Cells/L From Baseline	Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.	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)
Secondary	Number of Participants With Peak Platelet Counts of = 100 x 10? Cells/L	Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.	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)
Secondary	Number of Participants With Peak Platelet Counts of = 450 x 10? Cells/L	Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.	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)
Secondary	Change From Baseline to Peak Platelet Level	Platelet count data after the use of rescue medication were not included.	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)
Secondary	Time to Peak Platelet Count	Time from the date of study drug administration (day 1, 15 or 22) to the day of peak platelet count after each dose. Platelet count data after the use of rescue medication were not included.	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
Secondary	Duration Within the Targeted Therapeutic Range	Targeted therapeutic platelet level was defined as a platelet count that was double the baseline level and between 50 and 450 × 10? cells/L.; Platelet count data after the use of rescue medication were not included.	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

External Links

•   AmgenTrials clinical trials website
•   FDA-approved Drug Labeling