Experimental anemia drug trial halted after only 3 patients
NCT ID NCT04854889
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a low-dose chemotherapy drug (decitabine) in people with a severe type of anemia that did not improve with standard treatments. Only 3 people were enrolled before the study was stopped early. The goal was to see if the drug could improve blood cell counts and reduce the need for transfusions.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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3 people
The number who actually took part.
- Started
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Apr 2021
- Finished
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Aug 2022
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion criteria: Subjects eligible for enrollment in the study should meet all of following criteria: 1. Diagnosis of acquired aplastic confirmed by peripheral blood and bone-marrow examinations. 2. 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. 3. 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. 4. Age ≥12 years old. 5. An Eastern Cooperative Oncology Group performance status score of 0 to 2 at screening. 6. 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: 1. Diagnosis of inherited bone marrow failure disorders. 2. Bone marrow reticulin grade of ≥2. 3. Having a plan to take thrombopoietin (TPO) receptor agonists. 4. Having a plan to undergo hematopoietic stem cell transplantation within 1 year. 5. Subjects with hemolytic paroxysmal nocturnal hemoglobinuria clone. 6. Having abnormalities of cytogenetic abnormalities related to myelodysplastic syndrome except for +8 or 20q- or -Y. 7. Previous or concurrent active malignancies with chemoradiotherapy, except localized tumors diagnosed more than one year previously and treated surgically with curative intent. 8. Cytopenias secondary to any other non-hematological disorders (e.g., liver cirrhosis, active connective tissue disease, or chronic persistent infectious diseases). 9. Active infection not adequately responding to appropriate therapy. 10. Positive for anti-human immunodeficiency virus antibodies, or current infection of hepatitis B virus or hepatitis C virus at screening. 11. Concurrent condition of acute hemorrhage in gastrointestinal tract or respiratory tract, or central nervous system. 12. Dysfunction of liver: Alanine aminotransferase or aspartate aminotransferase or total bilirubin is more than 2.0 times the upper limit of laboratory normal range. 13. Dysfunction of renal: creatinine clear rate is less than 30ml/min. 14. Clinically significant dysfunction of heart: class Ⅲ or Ⅳ of the New York Heart Association classification. 15. Uncontrolled diabetes mellitus. 16. History of congestive heart failure, unstable angina pectoris, myocardial infarction, arterial or venous thrombosis within one year before enrollment. 17. Lactating or pregnant women or patients who have no intention of using oral contraceptives or birth control. 18. Subjects with psychiatric history or severe cerebrovascular disease with cognitive disorder. 19. Participating in other clinical trials within 4 weeks before enrollment. 20. Hypersensitivity to decitabine or its components. 21. A history of decitabine, azacitidine, or other demethylation agents. 22. 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). 23. Patients who are considered to be ineligible for the study by the investigator for reasons other than above.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Regenerative Medicine Center
Tianjin, Tianjin Municipality, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.