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

Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04870346
• Other study ID #: 531-005
• Status: Enrolling by invitation
• Start date: May 10, 2021
• Est. completion date: June 30, 2024

Study information

• Verified date: August 2023
• Source: Kyowa Kirin Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 45
• Est. completion date: June 30, 2024
• Est. primary completion date: June 30, 2024
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia

Locations

Country	Name	City	State
Japan	Kanazawa University, College of Medical Pharmaceutical and Health Sciences, School of Medicine, Department of Hematology	Kanazawa	Ishikawa Prefecture

Sponsors (1)

Lead Sponsor	Collaborator
Kyowa Kirin Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Hematological response at 2 years after the start of romiplostim treatment in Study 531-003/531-004		2 years
Primary	Hematological response at up to 5 years after the start of romiplostim treatment in Study 531-003/531-004		5 years
Secondary	The duration of hematological response, and the presence or absence of occurrence of new chromosomal abnormality or transformation to AML/MDS.	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	52, 78, 104, 130, 156, 182, 208, 234, 260 weeks