Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia

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

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.

Study Overview

• Status: Completed
• Conditions: Aplastic Anemia
• Intervention / Treatment: Drug: Romiplostim

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Japan
  • Tokyo
    • Shinagawa, Tokyo, Japan
      • NTT Medical Center Tokyo

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AMG531	Drug: Romiplostim; Subcutaneous administration of 0 to 20ug/kg for 6 months

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Rate of achievement of complete response (CR) or partial response (PR)	27 weeks post-dose

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Kyowa Kirin Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): December 3, 2019
• Primary Completion (Actual): October 7, 2021
• Study Completion (Actual): October 29, 2021

Study Registration Dates

• First Submitted: September 18, 2019
• First Submitted That Met QC Criteria: September 18, 2019
• First Posted (Actual): September 19, 2019

Study Record Updates

• Last Update Posted (Actual): May 26, 2022
• Last Update Submitted That Met QC Criteria: May 25, 2022
• Last Verified: May 1, 2022

More Information

Terms related to this study

• Keywords: romiplostim; aplastic anemia
• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic
• Other Study ID Numbers: 531-004

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No