Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 6 June 2022
• Main ID: NCT03957694
• Date of registration: 09/05/2019
• Prospective Registration: No
• Primary sponsor: Kyowa Kirin Co., Ltd.
• Public title: Study of AMG531(Romiplostim) in Patients With Aplastic Anemia
• Scientific title: A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy
• Date of first enrolment: April 25, 2019
• Target sample size: 17
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03957694
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Japan

Key inclusion & exclusion 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.



Age minimum: 20 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: Romiplostim

Primary Outcome(s)

Achievement of complete response (CR) or partial response (PR) [Time Frame: 27 weeks post-dose]

Secondary Outcome(s)

Change from baseline in platelet count (/µL) [Time Frame: Each time point evaluated weekly until Week 27]

Reduction or independence of platelet and/or erythrocyte transfusion [Time Frame: Week 27]

Change from baseline in neutrophil count (/µL) [Time Frame: Each time point evaluated weekly until Week 27]

Achievement of CR or PR [Time Frame: Week 14]

Change from baseline in hemoglobin (Hb) concentration (g/dL) [Time Frame: Each time point evaluated weekly until Week 27]

Change from baseline in reticulocyte count (/µL) [Time Frame: Each time point evaluated weekly until Week 27]

The time to CR or PR [Time Frame: Each time point evaluated weekly until Week 27]

Achievement of CR [Time Frame: Weeks 14 and 27]

Secondary ID(s)

531-003

Source(s) of Monetary Support

Please refer to primary and secondary sponsors