Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy

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.

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 17
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Japan
  • Ehime, Japan
    • Matsuyama Red Cross Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 20 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 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 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; Romiplostim SC. Initial dose is 10 ug/kg/. Maximum dose is 20 ug/kg. Original duration is 6 months from first administration but if investigator decide that the patient is needed more treatment, they move to extention period and take treatment up to 1 year.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Time Frame
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
Achievement of CR or PR	Week 14
Achievement of CR	Weeks 14 and 27

Collaborators and Investigators

• Sponsor: Kyowa Kirin Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): April 25, 2019
• Primary Completion (Actual): November 5, 2020
• Study Completion (Actual): May 26, 2021

Study Registration Dates

• First Submitted: May 9, 2019
• First Submitted That Met QC Criteria: May 19, 2019
• First Posted (Actual): May 21, 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: 531-003

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