Brief Title
Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia
Official Title
A Phase 2/3 Study of AMG531 Combined With Ciclosporin A in Patients With Aplastic Anemia Previously Untreated With Immunosuppressive Therapy
Brief Summary
To evaluate the hematological responses based on the response assessment criteria defined in
this study (the 531-004 response assessment criteria) when AMG531 is subcutaneously
(SC)-administered with ciclosporin A (CsA) therapy for 6 months in patients with aplastic
anemia (AA) who were previously untreated with immunosuppressive therapy.
Study Phase
Phase 2/Phase 3
Study Type
Interventional
Primary Outcome
Rate of achievement of complete response (CR) or partial response (PR)
Secondary Outcome
Rate of achievement of CR or PR
Condition
Aplastic Anemia
Intervention
Romiplostim
Study Arms / Comparison Groups
AMG531
Description:
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
24
Start Date
December 3, 2019
Completion Date
May 2022
Primary Completion Date
October 2021
Eligibility 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:
1. Previously treated with Anti-human thymocyte immunoglobulin (ATG), CsA, or
Alemtuzumab;
2. Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
3. Diagnosed as having acute myelocytic leukemia (AML) or chronic myelomonocytic
leukemia;
4. Concurrent thrombocytopenia of other etiologies (e.g., myelodysplastic syndrome (MDS),
idiopathic thrombocytopenic purpura (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 paroxysmal nocturnal hemoglobinuria (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.
Gender
All
Ages
20 Years - N/A
Accepts Healthy Volunteers
No
Contacts
, ,
Location Countries
Japan
Location Countries
Japan
Administrative Informations
NCT ID
NCT04095936
Organization ID
531-004
Responsible Party
Sponsor
Study Sponsor
Kyowa Kirin Co., Ltd.
Study Sponsor
, ,
Verification Date
June 2021