Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

NCT03957694 | Completed Phase 2

• 1 other identifier: 531-003
• Study Type: interventional
• Target: 17
• Locations: 1 country
• Sites: 1

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.

Conditions

Aplastic Anemia

Keywords

aplastic anemia; romiplostim

Outcome Measures

Primary Outcomes (1)

• Achievement of complete response (CR) or partial response (PR)

  27 weeks post-dose

Secondary Outcomes (8)

• Achievement of CR or PR

  Week 14

• 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

Study Arms (1)

AMG531

EXPERIMENTAL

Drug: Romiplostim

Interventions

Romiplostim DRUG

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.

AMG531

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Voluntary signed informed consent to participate in the study;
• A diagnosis of AA confirmed by blood and bone-marrow examinations, etc.;
• Considered to require new treatment with ATG and CsA provided that NSAA must be platelet or erythrocyte transfusion-dependent.
• An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score of 0 to 1at screening

You may not qualify if:

• Previously treated with ATG, CsA, or Alemtuzumab;
• Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
• Diagnosed as having AML or chronic myelomonocytic leukemia;
• Concurrent thrombocytopenia of other etiologies (e.g., MDS, ITP, cirrhosis);
• Concurrent active infection not adequately responding to appropriate therapy;
• Concurrent clinically significant illness(es) items which are deemed by the Investigator to be likely to affect the study conduct and assessments.
• Having active malignancies, or having a history of treatment of malignancies within 5 years prior to informed consent.
• Concurrent PNH
• Having Grade 2 or higher bone marrow reticulin based on Bone Marrow Pathology (2nd edition) ;
• History of chromosome aberrations discovered in bone marrow cells.
• Having blast cells \> 2% in bone marrow;
• Positive for anti-human immunodeficiency virus (HIV) antibody;
• Receiving prophylactic or therapeutic treatment for hepatitis type B
• Positive for hepatitis C virus (HCV) antibody, and HCV infection being confirmed
• Planned hematopoietic stem cell transplantation during the study;

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead

Study Sites (1)

Matsuyama Red Cross Hospital

Ehime, Japan

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: multi-national, open-label, phase 2/3 study

Study Record Dates

• First Submitted: May 9, 2019
• First Posted: May 21, 2019
• Study Start: April 25, 2019
• Primary Completion: November 5, 2020
• Study Completion: May 26, 2021
• Last Updated: May 26, 2022

Record last verified: 2022-05

Locations

JP (1)