Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia
- Registration Number
- NCT04854889
- Brief Summary
This is a phase 2, single-center, single-arm, open-label trial. Simon's two-stage design is performed to evaluate the efficacy of low-dose Decitabine in refractory aplastic anemia.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 3
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Decitabine Decitabine Subjects will receive low-dose decitabine for 4 cycles and for another 6 cycles in extension study for patients achieving response during the first 4 cycles.
- Primary Outcome Measures
Name Time Method Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) after 4 cycles of low-dose decitabine At 20 weeks
- Secondary Outcome Measures
Name Time Method Absolute changes in blood cell count Within 52 weeks Time from the first decitabine to hematologic response Within 20 weeks Duration of hematologic response Within 52 weeks Proportion of clonal evolution Within 52 weeks Incidence of drug-related adverse events Within 52 weeks Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) Within 52 weeks Proportion of subjects with transfusion independence or decreased transfusion requirement Within 52 weeks The utility score of EQ-5D-5L questionnaire Baseline, 20 weeks, 52 weeks Health-related quality of life is measure by the EQ-5D-5L questionnaire.
Proportion of relapse Within 52 weeks
Trial Locations
- Locations (1)
Regenerative Medicine Center
🇨🇳Tianjin, Tianjin, China