Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia

NCT ID: NCT04095936

Last Updated: 2022-05-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-12-03
• Study Completion Date: 2021-10-29

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.

Conditions

Aplastic Anemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

AMG531

Group Type: EXPERIMENTAL

Romiplostim

Intervention Type: DRUG

Subcutaneous administration of 0 to 20ug/kg for 6 months

Interventions

Romiplostim

Subcutaneous administration of 0 to 20ug/kg for 6 months

Intervention Type: DRUG

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.

• Minimum Eligible Age: 20 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kyowa Kirin Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

NTT Medical center Tokyo

Shinagawa, Tokyo, Japan

Countries

Japan

Other Identifiers

531-004

Identifier Type: -

Identifier Source: org_study_id