Aplastic Anemia
Conditions
Sponsors
Kyowa Kirin Co., Ltd
Eligibility
Sex/Gender
All
Inclusion criteria
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 immunosuppressive therapy 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
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) Having hepatitis C virus (HCV) infection confirmed by HCV-RNA or other tests at screening. 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: - 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| efficacy Achievement of complete response (CR) or partial response (PR) | — |
Secondary
| Measure | Time frame |
|---|---|
| efficacy - Achievement of CR or PR - The time to CR or PR - Maximum duration where response is continuously - Reduction or independence of platelet and/or erythrocyte transfusion - Change from baseline in platelet count, hemoglobin (Hb) concentration, neutrophil count, and reticulocyte count at each evaluation time point | — |
Countries
Asia except Japan, Japan
Outcome results
None listed