Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia

Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04095936
• Other study ID #: 531-004
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: December 3, 2019
• Est. completion date: October 29, 2021

Study information

• Verified date: May 2022
• Source: Kyowa Kirin Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: October 29, 2021
• Est. primary completion date: October 7, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia

Intervention

Drug:
• Romiplostim
Subcutaneous administration of 0 to 20ug/kg for 6 months

Locations

Country	Name	City	State
Japan	NTT Medical center Tokyo	Shinagawa	Tokyo

Sponsors (1)

Lead Sponsor	Collaborator
Kyowa Kirin Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rate of achievement of complete response (CR) or partial response (PR)		27 weeks post-dose
Secondary	Rate of achievement of CR or PR		14 weeks
Secondary	Rate of achievement of CR		Weeks 14 and 27
Secondary	The time to CR or PR		Each time point evaluated weekly until Week 27
Secondary	Reduction or independence of platelet and/or erythrocyte transfusion		Week 27
Secondary	Change from baseline in platelet count (/µL)		Each time point evaluated weekly until Week 27
Secondary	Change from baseline in hemoglobin (Hb) concentration (g/dL)		Each time point evaluated weekly until Week 27
Secondary	Change from baseline in neutrophil count (/µL)		Each time point evaluated weekly until Week 27
Secondary	Change from baseline in reticulocyte count (/µL)		Each time point evaluated weekly until Week 27