Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia

NCT02773290 | Completed Phase 2

• 1 other identifier: 531-002
• Study Type: interventional
• Target: 46
• Locations: 2 countries
• Sites: 3

Brief Summary

The objective of this study is to evaluate the efficacy of romiplostim administered once weekly to Aplastic Anemia (AA) patients with thrombocytopenia refractory to or ineligible for immunosuppressive therapy in Japan and Korea. Safety and pharmacokinetics of romiplostim after repeated administration will also be assessed.

Conditions

Aplastic Anemia

Keywords

Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) at Week 27

  At 27 weeks after dosing

Secondary Outcomes (4)

• Proportion of subjects with a hematological response at the end-of- treatment examination

  Up to 52 weeks after dosing

• Time from the first romiplostim administration to hematological response

  Up to 52 weeks after dosing

• In subjects receiving platelet transfusion as a pretreatment within 8 weeks prior to the first romiplostim administration; proportion of subjects with transfusion independence or decreased platelet transfusion requirement

  Up to 52 weeks after dosing

• Proportion of subjects achieving platelet response, erythroid response, or neutrophil response at each of Week 27 and end of treatment.

  At 27 weeks and 52 weeks after dosing

Study Arms (1)

Romiplostim

EXPERIMENTAL

Weekly Subcutaneous (SC) administration

Biological: Romiplostim

Interventions

Romiplostim BIOLOGICAL

Weekly SC administration

Romiplostim

Eligibility Criteria

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

You may qualify if:

• Diagnosis of Aplastic Anemia (AA) confirmed by peripheral blood and bone-marrow examinations, etc.
• Refractory to at least one course of immunosuppressive therapy including horse or rabbit anti-human thymocyte immunoglobulin (ATG); or ineligible for ATG treatment and refractory to cyclosporin (CyA)
• Thrombocytopenia defined as a platelet count of ≤ 30 × 10\^9/L
• Preserving main organ function as a result of screening as follows;
• Total bilirubin: \< 1.5 times the upper limit of the laboratory normal range
• Alanine aminotransferase: \< 3.0 times the upper limit of the laboratory normal range
• Aspartate aminotransferase: \< 3.0 times the upper limit of the laboratory normal range
• Creatinine value: ≤ 2.0 mg/dL
• An Eastern Cooperative Oncology Group performance status score of 0 to 2 at screening
• ≥ 20 years of age at the time of obtaining informed consent
• Patients who have provided written informed consent of their free will to participate in this study

You may not qualify if:

• Concurrent active infection not adequately responding to appropriate therapy
• Bone marrow reticulin grade of ≥ 2 based on the grading scale for reticulin indicated in Bone Marrow Pathology (2nd edition)
• Proportion of blasts in bone marrow \> 2%
• Previous or concurrent active malignancies, other than localized tumors diagnosed more than one year previously and treated surgically with curative intent (basal cell carcinoma; or surgically resected in situ carcinoma of the cervix with an apparent success of ≥ 12 months prior to enrollment; as well as other cancers which have not been treated and remained disease-free for at least 5 years before enrollment are eligible)
• Clinically significant cardiac disease (class III or IV of the New York Heart Association classification; unstable angina pectoris; myocardial infarction within 6 months before enrollment; cardiac disease accompanied by angioplasty or stenting within 6 months before enrollment; or clinically significant cardiac arrhythmias) or uncontrollable hypertension
• Arterial or venous thrombosis within one year before enrollment
• Positive for anti-human immunodeficiency virus antibodies, hepatitis B surface antigen, or hepatitis C virus-RNA at screening
• Thrombocytopenia due to any other cause (e.g., myelodysplastic syndrome, idiopathic thrombocytopenic purpura, or liver cirrhosis)
• Patients with acute myeloblastic leukemia or chronic myelomonocytic leukemia
• Concurrent occurrence of hemolytic predominant paroxysmal nocturnal hemoglobinuria (Hemolytic predominant is defined as lactate dehydrogenase \> 1.5 times the upper limit of the laboratory normal range)
• Uncontrolled diabetes mellitus
• Receiving other investigational products within 16 weeks before romiplostim treatment initiation
• Receiving any agent to treat AA, including the following agents before romiplostim treatment initiation;
• ATG treatment within 6 months before romiplostim treatment initiation
• CyA or anabolic steroid treatment within 6 weeks before romiplostim treatment initiation:

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead

Study Sites (3)

Unknown Facility

Kanazawa, Japan

Location

Unknown Facility

Tokyo, Japan

Location

Unknown Facility

Seoul, South Korea

Location

Related Publications (1)

• Jang JH, Mitani K, Tomiyama Y, Miyazaki K, Nagafuji K, Usuki K, Uoshima N, Fujisaki T, Kosugi H, Matsumura I, Sasaki K, Kizaki M, Sawa M, Hidaka M, Kobayashi N, Ichikawa S, Yonemura Y, Murotani K, Shimizu M, Matsuda A, Ozawa K, Nakao S, Lee JW. Predictive factors of romiplostim response in patients with refractory aplastic anemia: data from two clinical trials. Ann Hematol. 2025 Aug;104(8):4003-4011. doi: 10.1007/s00277-025-06337-7. Epub 2025 Jul 1.

  PMID: 40593138 DERIVED

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

Study Record Dates

• First Submitted: May 9, 2016
• First Posted: May 16, 2016
• Study Start: May 1, 2016
• Primary Completion: November 14, 2017
• Study Completion: July 28, 2020
• Last Updated: July 16, 2024

Record last verified: 2024-07

Data Sharing

• IPD Sharing: Will not share

Locations

JP (2); KR (1)