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

NCT04870346 | Active Not Recruiting

• 1 other identifier: 531-005
• Study Type: observational
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Aplastic Anemia

Keywords

aplastic anemia

Outcome Measures

Primary Outcomes (2)

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

  2 years

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

  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.

  52, 78, 104, 130, 156, 182, 208, 234, 260 weeks

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients enrolled in Study 531-003/531-004

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead

Study Sites (1)

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

Kanazawa, Ishikawa-ken, 920-0934, Japan

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

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

Study Officials

• Hirohito Yamazaki

  Kanazawa University Hospital, Blood Transfusion Department

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 22, 2021
• First Posted: May 3, 2021
• Study Start: May 10, 2021
• Primary Completion: August 31, 2024
• Study Completion (Estimated): June 30, 2027
• Last Updated: September 11, 2025

Record last verified: 2025-09

Locations

JP (1)