Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

Brief Title

Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

Official Title

A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy

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.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional

Primary Outcome

Achievement of complete response (CR) or partial response (PR)

Secondary Outcome

 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 Status

Drug

Estimated Enrollment

17

Start Date

April 25, 2019

Completion Date

May 26, 2021

Primary Completion Date

November 5, 2020

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 ATG and CsA 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 ATG, CsA, or Alemtuzumab;

          2. Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);

          3. Diagnosed as having AML or chronic myelomonocytic leukemia;

          4. Concurrent thrombocytopenia of other etiologies (e.g., MDS, 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 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. Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed

         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, however, excluding their use as premedication:

               -  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

NCT ID

NCT03957694

Organization ID

531-003

Responsible Party

Sponsor

Study Sponsor

Kyowa Kirin Co., Ltd.

Study Sponsor

, , 

Verification Date

May 2022