Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia (NCT04854889) | Clinical Trial Compass
TerminatedPhase 2
Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia
Stopped: Not achieve the desired effect
China3 participantsStarted 2021-04-22
Plain-language 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.
Who can participate
Age range12 Years
SexALL
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Inclusion criteria
✓. Diagnosis of acquired aplastic confirmed by peripheral blood and bone-marrow examinations.
✓. Refractory to standard first-line immunosuppressive therapy for at least 6 months, including the combination of rabbit anti-thymocyte globulin (rATG) or porcine anti-lymphocyte globulin (pALG) and cyclosporine (CsA); or ineligible for rATG/ pALG and refractory to CsA alone.
✓. Persistent decrease of blood cell count, including platelet \<30×10\^9/L, and/or hemoglobin \<90g/L, and/or absolute neutrophil count \<0.5×10\^9/L.
✓. Age ≥12 years old.
✓. An Eastern Cooperative Oncology Group performance status score of 0 to 2 at screening.
✓. Patients or their legally authorized representatives who have provided written informed consent of their free will to participate in this study.
Exclusion criteria
✕. Diagnosis of inherited bone marrow failure disorders.
✕. Bone marrow reticulin grade of ≥2.
✕. Having a plan to take thrombopoietin (TPO) receptor agonists.
✕. Having a plan to undergo hematopoietic stem cell transplantation within 1 year.
✕. Subjects with hemolytic paroxysmal nocturnal hemoglobinuria clone.
✕. Having abnormalities of cytogenetic abnormalities related to myelodysplastic syndrome except for +8 or 20q- or -Y.
What they're measuring
1
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) after 4 cycles of low-dose decitabine
Timeframe: At 20 weeks
Trial details
NCT IDNCT04854889
SponsorInstitute of Hematology & Blood Diseases Hospital, China
✕. Previous or concurrent active malignancies with chemoradiotherapy, except localized tumors diagnosed more than one year previously and treated surgically with curative intent.
✕. Cytopenias secondary to any other non-hematological disorders (e.g., liver cirrhosis, active connective tissue disease, or chronic persistent infectious diseases).