Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia

NCT04854889 | Terminated Phase 2

• 1 other identifier: IIT2021016
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

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.

Conditions

Refractory Aplastic Anemia

Outcome Measures

Primary Outcomes (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

  At 20 weeks

Secondary Outcomes (9)

• 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

• Absolute changes in blood cell count

  Within 52 weeks

• The utility score of EQ-5D-5L questionnaire

  Baseline, 20 weeks, 52 weeks

Study Arms (1)

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.

Drug: Decitabine

Interventions

Decitabine DRUG

Demethylating agents

Decitabine

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Decitabine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 14, 2021
• First Posted: April 22, 2021
• Study Start: April 22, 2021
• Primary Completion: July 26, 2022
• Study Completion: August 9, 2022
• Last Updated: February 11, 2026

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)