Status:
TERMINATED
Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia
Lead Sponsor:
Institute of Hematology & Blood Diseases Hospital, China
Conditions:
Refractory Aplastic Anemia
Eligibility:
All Genders
12+ years
Phase:
PHASE2
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.
Eligibility Criteria
Inclusion
- Inclusion criteria:
- Subjects eligible for enrollment in the study should meet all of following 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:
- Subjects must be excluded from participating in this study if they meet any of the following 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.
- 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).
- Active infection not adequately responding to appropriate therapy.
- Positive for anti-human immunodeficiency virus antibodies, or current infection of hepatitis B virus or hepatitis C virus at screening.
- Concurrent condition of acute hemorrhage in gastrointestinal tract or respiratory tract, or central nervous system.
- Dysfunction of liver: Alanine aminotransferase or aspartate aminotransferase or total bilirubin is more than 2.0 times the upper limit of laboratory normal range.
- Dysfunction of renal: creatinine clear rate is less than 30ml/min.
- Clinically significant dysfunction of heart: class Ⅲ or Ⅳ of the New York Heart Association classification.
- Uncontrolled diabetes mellitus.
- History of congestive heart failure, unstable angina pectoris, myocardial infarction, arterial or venous thrombosis within one year before enrollment.
- Lactating or pregnant women or patients who have no intention of using oral contraceptives or birth control.
- Subjects with psychiatric history or severe cerebrovascular disease with cognitive disorder.
- Participating in other clinical trials within 4 weeks before enrollment.
- Hypersensitivity to decitabine or its components.
- A history of decitabine, azacitidine, or other demethylation agents.
- Receiving TPO-receptor agonists within 1 month before enrollment (other than patients who are treated with TPO-receptor agonists and have no response after at least 4 months treatment).
- Patients who are considered to be ineligible for the study by the investigator for reasons other than above.
Exclusion
Key Trial Info
Start Date :
April 22 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 9 2022
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT04854889
Start Date
April 22 2021
End Date
August 9 2022
Last Update
August 30 2022
Active Locations (1)
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1
Regenerative Medicine Center
Tianjin, Tianjin Municipality, China