A Phase 2 interventional study of Tacrolimus and Placebo (for Tacrolimus) in Aplastic Anemia and Drug Effect, sponsored by Peking Union Medical College Hospital. Completed at 1 site in China. Open to participants aged 14 Years to 85 Years. Per ClinicalTrials.gov, last updated 2023-01-19.
Sponsored by Peking Union Medical College Hospital · Phase 2, Interventional, and Treatment
This is a randomized, open-label, phase II study to compare the efficacy of eltrombopag combined with tacrolimus to eltrombopag alone in Chinese subjects with refractory or relapsed aplastic anemia. The safety would also be evaluated. Patients would be randomized to receive eltrombopag alone or eltrombopag combined with tacrolimus. Treatment with eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Tacrolimus will be given at 1mg bid with the target trough concentration of 4-10 ng/mL throughout the study. The hematological response rate and safety will be recorded and compared at 3, 6 months and 1 year after starting the study treatment (Week 13, 26 and 52).
1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.
This study's enrollment of 114 is above the median of 94 across 1,291 interventional studies indexed under Anemia.
Browse Anemia studies →Peking Union Medical College Hospital is the lead sponsor of 1,115 studies on the registry; 463 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Eltrombopag and the placebo would be applied. Eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved.
Drug: Placebo (for Tacrolimus)
Eltrombopag and tacrolimus would be applied. Eltrombopag will be started at 25 mg/day and increased by 25 mg/day every 2 weeks according to the platelet count up to 150 mg/day, or the best response was achieved. Tacrolimus will be given at 1mg bid with the target trough concentration of 4-10 ng/mL throughout the study.
Drug: Tacrolimus
Tacrolimus will be given at 1mg bid with the target trough concentration to be 4-10 ng/mL.
Also known as: experimental
placebo will be given at 1mg bid.
Also known as: control
ORR at 6 Months
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at Week 26
Time frame: Week 26
ORR at 3 Months
ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.
Time frame: Week 14
Changes in Haemoglobin in the Absence of Red Blood Cells Transfusion
The change in hematology values ( haemoglobin) were evaluated
Time frame: Week 26
Changes in Platelet in the Absence of Platelet Transfusion
The change in hematology values (platelet) were evaluated
Time frame: Week 26
Duration of hematologic response
Time from the date of the start of the first response to the date of first relapse defined as again meeting criteria for aplastic anemia
Time frame: by 6 months (all patients), at 24 months (responders only)
Percentage of patients with clonal evolution to myelodysplasia, PNH, acute leukemia
Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline \< 10% that rose to greater than 50% on study.
Time frame: 12 months
Plan to share: Yes — individual participant data would be accepted upon request
Supporting information: Study protocol, Sap, Icf, Csr
No publications or documents are linked to this record.
This study is completed, as verified in Jan 2023. You cannot join it, but the record below documents what was studied.
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Peking Union Medical College Hospital