Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia

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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