Overview

This trial is active, not recruiting.

Condition aplastic anemia
Treatment amg531
Phase phase 2
Sponsor Kyowa Hakko Kirin Korea Co., Ltd.
Start date March 2014
End date September 2017
Trial size 32 participants
Trial identifier NCT02094417, 531-KR001

Summary

The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the Aplastic Anemia (AA) patients with immunosuppressive-therapy refractory thrombocytopenia and also to assess the pharmacokinetics of this product. Its efficacy and safety during the extension period beyond one year will also be evaluated.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety/efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
amg531
Subcutaneous, weekly injection
(Experimental)
amg531
Subcutaneous, weekly injection
(Experimental)
amg531
Subcutaneous, weekly injection
(Experimental)
amg531
Subcutaneous, weekly injection

Primary Outcomes

Measure
The proportion of subjects achieving a platelet response
time frame: At week 9

Secondary Outcomes

Measure
The proportion of subjects achieving a platelet response
time frame: 52 weeks
The proportion of subjects who become platelet transfusion independent
time frame: 156 weeks
The proportion of subjects achieving erythroid response
time frame: 156 weeks
The proportion of subjects achieving neutrophil response
time frame: 156 Weeks
Changes in Gruppo Italiano Malattie Ematologiche Maligne dell' Adulto (GIMEMA) bleeding scale
time frame: 156 weeks
Profiles of Pharmacokinetics
time frame: Weeks 1, 4, and 8
Incidences of adverse events
time frame: 156 weeks + 4 weeks follow-up
Development of neutralizing antibody
time frame: 156 weeks + 4 weeks follow-up

Eligibility Criteria

Male or female participants at least 19 years old.

Inclusion Criteria: - Patient who is diagnosed as AA and refractory to immunosuppressive therapy - Platelet ≤ 30,000/μL Exclusion Criteria: - Concurrent active infection not adequately responding to appropriate therapy - HIV positivity - Bone marrow reticulin grade of > 1 - Clinically significant cardiac disease - Arterial or venous thrombosis within the last 1 year before enrollment - Other cause of thrombocytopenia - AA with hemolytic predominant paroxysmal nocturnal hemoglobinuria (PNH) - Uncontrolled diabetes - Receiving any agent used to treat AA, including antithymocyte globulin (ATG) or ATG + cyclosporine within 6 months before starting study treatment and/or cyclosporine or anabolic hormone within 6 weeks before starting the study treatment - History of PEG-rHuMGDF, recombinant human thrombopoietin, AMG531, and other thrombopoietin (TPO)-receptor agonist - Who plans to conduct hematopoietic stem cell transplantation within 1 year

Additional Information

Official title A Phase 2, Randomized, Open-Label, Parallel, Comparative, Dose-Finding Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia Subjects With Thrombocytopenia Refractory to Immunosuppressive Therapy
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by Kyowa Hakko Kirin Korea Co., Ltd..