Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT04870346

Long Term Follow-up Observational Study After Clinical Trials of AMG531 (Romiplostim) in Patients With Untreated Aplastic Anemia

To evaluate the long-term efficacy and safety after the end of romiplostim treatment by observation [up to 5 years] of patients who were registered for Study 531-003/531-004 in immunosuppressive therapy-naïve patients with aplastic anemia.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Kanazawa University, College of Medical Pharmaceutical and Health Sciences, School of Medicine, Department of Hematology

Kanazawa, Ishikawa-ken, 920-0934, Japan

Who can participate

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients whose written consent to participate in this research was obtained among patients registered for Study 531-003/531-004
  • Of the patients who died before their consent to participate in this research was obtained among those registered for Study 531-003/531-004, patients whose legally authorized representatives such as their family had given the consent or patients whose legally authorized representatives had not refused in the disclosure of information by opt-out
  • Of the patients who died before their consent to participate in this research was obtained among those registered for Study 531-003/531-004, patients, patients for whom the consent process exemption was approved by the Ethics Review Committee, etc. of the participating medical institutions at the participating medical institutions in the countries without the opt-out system

Exclusion criteria

  • Patients who are judged by the investigator or sub-investigator to be unfavorable for participation in this research

Treatment and study plan

Primary outcomes

  1. Hematological response at 2 years after the start of romiplostim treatment in Study 531-003/531-004

    Time frame: 2 years

  2. Hematological response at up to 5 years after the start of romiplostim treatment in Study 531-003/531-004

    Time frame: 5 years

Secondary outcomes

  1. The duration of hematological response, and the presence or absence of occurrence of new chromosomal abnormality or transformation to AML/MDS.

    Time frame: 52, 78, 104, 130, 156, 182, 208, 234, 260 weeks

    • Duration of hematological response in patients who achieved hematologic response at the completion of Study 531-003/531-004
    • Time courses of Hb level (g/dL)
    • Time courses of platelet count (/μL)
    • Time courses of neutrophil count (/μL)
    • Presence or absence of platelet blood cell transfusion
    • Presence or absence of red blood cell transfusion
    • Presence or absence of G-CSF product administration
    • Dose level and administration period of cyclosporine A
    • Presence or absence of transformation to MDS/AML
    • Presence or absence of occurrence of new chromosome abnormality
    • Time to the start of subsequent treatment or to death
    • Overall survival

Sponsors and collaborators

Lead sponsor

Kyowa Kirin Co., Ltd.

Industry

Registry information

Important dates

Study start
2021
Primary completion
2024
Study completion
2027
First posted
May 3, 2021
Registry last updated
Sep 11, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.