Long Term Follow-up Observational Study After Clinical Trials of AMG531 (Romiplostim) in Patients With Untreated Aplastic Anemia
- Conditions
- Aplastic Anemia
- Registration Number
- NCT04870346
- Lead Sponsor
- Kyowa Kirin Co., Ltd.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 36
- 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
- Patients who are judged by the investigator or sub-investigator to be unfavorable for participation in this research
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Hematological response at up to 5 years after the start of romiplostim treatment in Study 531-003/531-004 5 years Hematological response at 2 years after the start of romiplostim treatment in Study 531-003/531-004 2 years
- Secondary Outcome Measures
Name Time Method 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 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
Trial Locations
- Locations (1)
Kanazawa University, College of Medical Pharmaceutical and Health Sciences, School of Medicine, Department of Hematology
🇯🇵Kanazawa, Ishikawa Prefecture, Japan