Aplastic Anemia
Conditions
Keywords
aplastic anemia, romiplostim
Brief summary
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.
Interventions
DRUGRomiplostim
Romiplostim SC. Initial dose is 10 ug/kg/. Maximum dose is 20 ug/kg. Original duration is 6 months from first administration but if investigator decide that the patient is needed more treatment, they move to extention period and take treatment up to 1 year.
Sponsors
Kyowa Kirin Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
multi-national, open-label, phase 2/3 study
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Achievement of complete response (CR) or partial response (PR) | 27 weeks post-dose |
Secondary
| Measure | Time frame |
|---|---|
| Achievement of CR | Weeks 14 and 27 |
| The time to CR or PR | Each time point evaluated weekly until Week 27 |
| Reduction or independence of platelet and/or erythrocyte transfusion | Week 27 |
| Achievement of CR or PR | Week 14 |
| Change from baseline in hemoglobin (Hb) concentration (g/dL) | Each time point evaluated weekly until Week 27 |
| Change from baseline in neutrophil count (/µL) | Each time point evaluated weekly until Week 27 |
| Change from baseline in reticulocyte count (/µL) | Each time point evaluated weekly until Week 27 |
| Change from baseline in platelet count (/µL) | Each time point evaluated weekly until Week 27 |
Countries
Japan
Outcome results
None listed