A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia
- Conditions
- Aplastic Anemia
- Registration Number
- JPRN-jRCT2080224878
- Lead Sponsor
- Kyowa Kirin Co., Ltd
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 24
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;
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method efficacy<br>Achievement of complete response (CR) or partial response (PR)
- Secondary Outcome Measures
Name Time Method efficacy<br>- Achievement of CR or PR <br>- The time to CR or PR <br>- Maximum duration where response is continuously <br>- Reduction or independence of platelet and/or erythrocyte transfusion<br>- Change from baseline in platelet count, hemoglobin (Hb) concentration, neutrophil count, and reticulocyte count at each evaluation time point