Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2/Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: AMG531
|
Drug: Romiplostim
Subcutaneous administration of 0 to 20ug/kg for 6 months
|
Outcome Measures
Primary Outcome Measures
- Rate of achievement of complete response (CR) or partial response (PR) [27 weeks post-dose]
Secondary Outcome Measures
- Rate of achievement of CR or PR [14 weeks]
- Rate of 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]
- Change from baseline in platelet count (/µL) [Each time point evaluated weekly until Week 27]
- 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]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Voluntary signed informed consent to participate in the study;
-
A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
-
Considered to require new treatment with immunosuppressive therapy provided that NSAA must be platelet or erythrocyte transfusion-dependent.
-
An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening;
Exclusion Criteria:
-
Previously treated with Anti-human thymocyte immunoglobulin (ATG), CsA, or Alemtuzumab;
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Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
-
Diagnosed as having acute myelocytic leukemia (AML) or chronic myelomonocytic leukemia;
-
Concurrent thrombocytopenia of other etiologies (e.g., myelodysplastic syndrome (MDS), idiopathic thrombocytopenic purpura (ITP), cirrhosis);
-
Concurrent active infection not adequately responding to appropriate therapy;
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Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments.
-
Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
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Concurrent paroxysmal nocturnal hemoglobinuria (PNH)
-
Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;
-
History of chromosome aberrations discovered in bone marrow cells.
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Having blast cells > 2% in bone marrow;
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Positive for anti-human immunodeficiency virus (HIV) antibody;
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Receiving prophylactic or therapeutic treatment for hepatitis type B
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Having hepatitis C virus (HCV) infection confirmed by HCV-RNA or other tests at screening.
-
Planned hematopoietic stem cell transplantation during the study;
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Systemic treatment with any of the following medication for the treatment of AA within 4 weeks before Day 1:
-
Anabolic steroids
-
Corticosteroids;
-
Pregnant or breastfeeding women, or women willing to become pregnant;
-
Other conditions unsuitable for participation in the study in the opinion of the Investigator.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | NTT Medical center Tokyo | Shinagawa | Tokyo | Japan |
Sponsors and Collaborators
- Kyowa Kirin Co., Ltd.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 531-004