Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

Phase 2

Completed

• Conditions: Aplastic Anemia
• Interventions: Drug: Romiplostim

• Registration Number: NCT03957694
• Lead Sponsor: Kyowa Kirin Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-04-25
• Study First Submitted: 2019-05-09
• Study First Submitted QC: 2019-05-19
• Study First Posted: 2019-05-21
• Primary Completion: 2020-11-05
• Study Completion: 2021-05-26
• Status Verified: 2022-05
• Last Update Submitted: 2022-05-25
• Last Update Posted: 2022-05-26

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
AMG531	Romiplostim	-

Primary Outcome Measures

Name	Time	Method
Achievement of complete response (CR) or partial response (PR)	27 weeks post-dose

Secondary Outcome Measures

Name	Time	Method
Change from baseline in hemoglobin (Hb) concentration (g/dL)	Each time point evaluated weekly until Week 27
Achievement of CR	Weeks 14 and 27
Change from baseline in platelet count (/µL)	Each time point evaluated weekly until Week 27
Reduction or independence of platelet and/or erythrocyte transfusion	Week 27
Achievement of CR or PR	Week 14
The time to CR or PR	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

Trial Locations

Locations (1)

Matsuyama Red Cross Hospital; 🇯🇵 Ehime, Japan