Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia

NCT04095936 | Completed Phase 2

• 1 other identifier: 531-004
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Aplastic Anemia

Keywords

aplastic anemia; romiplostim

Outcome Measures

Primary Outcomes (1)

• Rate of achievement of complete response (CR) or partial response (PR)

  27 weeks post-dose

Secondary Outcomes (8)

• 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

Study Arms (1)

AMG531

EXPERIMENTAL

Drug: Romiplostim

Interventions

Romiplostim DRUG

Subcutaneous administration of 0 to 20ug/kg for 6 months

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 immunosuppressive therapy 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 Anti-human thymocyte immunoglobulin (ATG), CsA, or Alemtuzumab;
• Diagnosed as having congenital AA (Fanconi anemia, congenital dyskeratosis, etc.);
• Diagnosed as having acute myelocytic leukemia (AML) or chronic myelomonocytic leukemia;
• Concurrent thrombocytopenia of other etiologies (e.g., myelodysplastic syndrome (MDS), idiopathic thrombocytopenic purpura (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 paroxysmal nocturnal hemoglobinuria (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
• Having hepatitis C virus (HCV) infection confirmed by HCV-RNA or other tests at screening.
• Planned hematopoietic stem cell transplantation during the study;

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead

Study Sites (1)

NTT Medical center Tokyo

Shinagawa, Tokyo, 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: September 18, 2019
• First Posted: September 19, 2019
• Study Start: December 3, 2019
• Primary Completion: October 7, 2021
• Study Completion: October 29, 2021
• Last Updated: May 26, 2022

Record last verified: 2022-05

Locations

JP (1)