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 range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2022-07-26

View on ClinicalTrials.gov More Refractory Aplastic Anemia trials

Who can participate

Age range

12 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

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

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

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

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria