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 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2/Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: AMG531
|
Drug: Romiplostim
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.
|
Outcome Measures
Primary Outcome Measures
- Achievement of complete response (CR) or partial response (PR) [27 weeks post-dose]
Secondary Outcome Measures
- Achievement of CR or PR [Week 14]
- 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;
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A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
-
Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent.
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An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening
Exclusion Criteria:
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Previously treated with ATG, CsA, or Alemtuzumab;
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Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
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Diagnosed as having AML or chronic myelomonocytic leukemia;
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Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis);
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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.
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Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
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Concurrent PNH
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Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;
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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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Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed
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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, however, excluding their use as premedication:
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Anabolic steroids
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Corticosteroids;
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Pregnant or breastfeeding women, or women willing to become pregnant;
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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 | Matsuyama Red Cross Hospital | Ehime | Japan |
Sponsors and Collaborators
- Kyowa Kirin Co., Ltd.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 531-003