Study of AMG531(Romiplostim) in Patients With Aplastic Anemia
A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy
To evaluate the hematological responses based on the response assessment criteria when AMG531 is subcutaneous (SC)-administered with anti-human thymocyte immunoglobulin (ATG) + ciclosporin A (CsA) therapy for 6 months in patients with aplastic anemia (AA) who were previously untreated with immunosuppressive therapy.
Phase 2/Phase 3
Achievement of complete response (CR) or partial response (PR)
Achievement of CR or PR
Study Arms / Comparison Groups
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
April 25, 2019
May 26, 2021
Primary Completion Date
November 5, 2020
Inclusion Criteria: 1. Voluntary signed informed consent to participate in the study; 2. A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.; 3. Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent. 4. An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening Exclusion Criteria: 1. Previously treated with ATG, CsA, or Alemtuzumab; 2. Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.); 3. Diagnosed as having AML or chronic myelomonocytic leukemia; 4. Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis); 5. Concurrent active infection not adequately responding to appropriate therapy; 6. Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments. 7. Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent. 8. Concurrent PNH 9. Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ; 10. History of chromosome aberrations discovered in bone marrow cells. 11. Having blast cells > 2% in bone marrow; 12. Positive for anti-human immunodeficiency virus (HIV) antibody; 13. Receiving prophylactic or therapeutic treatment for hepatitis type B 14. Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed 15. Planned hematopoietic stem cell transplantation during the study; 16. Systemic treatment with any of the following medication for the treatment of AA within 4 weeks before Day 1, however, excluding their use as premedication: - Anabolic steroids - Corticosteroids; 17. Pregnant or breastfeeding women, or women willing to become pregnant; 18. Other conditions unsuitable for participation in the study in the opinion of the Investigator.
20 Years - N/A
Accepts Healthy Volunteers
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