A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia

Kyowa Kirin

Status and phase

Completed

Phase 2

Conditions

Aplastic Anemia

Treatments

Drug: AMG531

Study type

Interventional

Funder types

Industry

Identifiers

NCT02094417

531-KR001

Details and patient eligibility

About

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.

Enrollment

35 patients

Sex

All

Ages

19+ years old

Volunteers

No Healthy Volunteers

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