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

August 4, 2023 updated by: Kyowa Kirin Co., Ltd.
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.

Study Overview

Status

Enrolling by invitation

Conditions

Study Type

Observational

Enrollment (Estimated)

45

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Japan
    • Ishikawa Prefecture
      • Kanazawa, Ishikawa Prefecture, Japan, 920-0934
        • Kanazawa University, College of Medical Pharmaceutical and Health Sciences, School of Medicine, Department of Hematology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

Patients enrolled in Study 531-003/531-004

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Hematological response at 2 years after the start of romiplostim treatment in Study 531-003/531-004
Time Frame: 2 years
2 years
Hematological response at up to 5 years after the start of romiplostim treatment in Study 531-003/531-004
Time Frame: 5 years
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
  1. Duration of hematological response in patients who achieved hematologic response at the completion of Study 531-003/531-004
  2. Time courses of Hb level (g/dL)
  3. Time courses of platelet count (/μL)
  4. Time courses of neutrophil count (/μL)
  5. Presence or absence of platelet blood cell transfusion
  6. Presence or absence of red blood cell transfusion
  7. Presence or absence of G-CSF product administration
  8. Dose level and administration period of cyclosporine A
  9. Presence or absence of transformation to MDS/AML
  10. Presence or absence of occurrence of new chromosome abnormality
  11. Time to the start of subsequent treatment or to death
  12. Overall survival
52, 78, 104, 130, 156, 182, 208, 234, 260 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Kyowa Kirin Co., Ltd.

Investigators

  • Principal Investigator: Hirohito Yamazaki, Kanazawa University Hospital, Blood Transfusion Department

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 10, 2021

Primary Completion (Estimated)

June 30, 2024

Study Completion (Estimated)

June 30, 2024

Study Registration Dates

First Submitted

April 22, 2021

First Submitted That Met QC Criteria

April 28, 2021

First Posted (Actual)

May 3, 2021

Study Record Updates

Last Update Posted (Actual)

August 7, 2023

Last Update Submitted That Met QC Criteria

August 4, 2023

Last Verified

August 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 531-005

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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