NCT04854889TerminatedPhase 2
Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia (LODACA): a Phase 2, Single-arm, Open-label Study
Institute of Hematology & Blood Diseases Hospital, China1 site in 1 country3 target enrollmentStarted: April 22, 2021Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 2
- Status
- Terminated
- Enrollment
- 3
- Locations
- 1
- Primary Endpoint
- Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) after 4 cycles of low-dose decitabine
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 12 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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 ≥
- •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.
Arms & Interventions
Decitabine
Experimental
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.
Intervention: Decitabine (Drug)
Outcomes
Primary Outcomes
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) after 4 cycles of low-dose decitabine
Time Frame: At 20 weeks
Secondary Outcomes
- 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)
- Proportion of relapse(Within 52 weeks)
Investigators
Jun Shi
Director of Regenerative Medicine Clinic Center
Institute of Hematology & Blood Diseases Hospital, China
Study Sites (1)
Loading locations...
Similar Trials
Completed
Phase 2
Study of Low-Dose Intravenous Decitabine in Patients Aged > 60 Years With Acute Myeloid LeukemiaAcute Myeloid LeukemiaNCT00866073University Hospital Freiburg238
Not yet recruiting
Phase 2
Decitabine Plus Penpulimab as Second-line Therapy for Advanced ESCC Treated With PD-1 BlockadeEsophageal Squamous Cell CarcinomaNCT05265962The First Affiliated Hospital of Zhengzhou University85
Completed
Phase 3
An Effectiveness and Safety Study of Decitabine in Patients With Myelodysplastic SyndromeMyelodysplastic SyndromeNCT01751867Xian-Janssen Pharmaceutical Ltd.135
Terminated
Phase 1
Parallel Phase I/II Trial of Decitabine and Peg-Interferon in Melanoma: Phase I PortionMelanomaNCT00791271M.D. Anderson Cancer Center17
Recruiting
Phase 2
Disitamab Vedotin Plus Cadonilimab in Patients With HER2 Mutant Advanced or Metastatic Bile Duct AdenocarcinomaBile Duct Adenocarcinoma Non-ResectableHER2 Gene MutationNCT06383533Zhejiang Cancer Hospital28