Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia

Study Overview
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Brief Summary
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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.

Official Title
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A Phase 2/3 Study of AMG531 Combined With Ciclosporin A in Patients With Aplastic Anemia Previously Untreated With Immunosuppressive Therapy

Conditions
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Aplastic Anemia

Intervention / Treatment
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* Drug: Romiplostim


Participation Criteria
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Eligibility Criteria
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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; 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; 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. 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. Having blast cells > 2% in bone marrow; Positive for anti-human immunodeficiency virus (HIV) antibody; Receiving prophylactic or therapeutic treatment for hepatitis type B Having hepatitis C virus (HCV) infection confirmed by HCV-RNA or other tests at screening. Planned hematopoietic stem cell transplantation during the study; 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.

Ages Eligible for Study
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Minimum Age: 20 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: multi-national, open-label, phase 2/3 study
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: AMG531<br> | Drug: Romiplostim<br>* Subcutaneous administration of 0 to 20ug/kg for 6 months<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of achievement of complete response (CR) or partial response (PR) |  | 27 weeks post-dose | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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aplastic anemia, romiplostim