Refractory Aplastic AnemiaPhase 2LODACANCT04854889
Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia
A Phase 2 study testing Decitabine in people with Refractory Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Refractory Aplastic Anemia
- What is being tested?
- Decitabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteChina
- Study dates
- 22 Apr 2021to 9 Aug 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia (LODACA): a Phase 2, Single-arm, Open-label Study
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
- Being tested
- Decitabine
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DecitabineDrug
Demethylating agents
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response) after 4 cycles of low-dose decitabine
Measured: At 20 weeks
Secondary outcomes (9)
Incidence of drug-related adverse events
Measured: Within 52 weeks
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response)
Measured: Within 52 weeks
Proportion of subjects with transfusion independence or decreased transfusion requirement
Measured: Within 52 weeks
Absolute changes in blood cell count
Measured: Within 52 weeks
The utility score of EQ-5D-5L questionnaire
Health-related quality of life is measure by the EQ-5D-5L questionnaire.
Measured: Baseline, 20 weeks, 52 weeks
Time from the first decitabine to hematologic response
Measured: Within 20 weeks
Duration of hematologic response
Measured: Within 52 weeks
Proportion of relapse
Measured: Within 52 weeks
Proportion of clonal evolution
Measured: Within 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Apr 2021
- Study start22 Apr 2021
- Primary completion26 Jul 2022
- Study completion9 Aug 2022
Why it stopped: Not achieve the desired effect
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Refractory Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04854889
- Sponsor's protocol ID
- IIT2021016
- Acronym
- LODACA
- Status last verified
- Aug 2022
- First submitted
- 14 Apr 2021
- Last updated
- 11 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Low-dose Decitabine in Refractory Aplastic Anemia (LODACA): a Phase 2, Single-arm, Open-label Study
- ClinicalTrials.gov ID
- NCT04854889
- Acronym / sponsor ID
- LODACA / IIT2021016
- Conditions
- Refractory Aplastic Anemia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Not achieve the desired effect
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 22 Apr 2021 (actual)
- Primary completion
- 26 Jul 2022 (actual)
- Study completion
- 9 Aug 2022 (actual)
- First posted
- 22 Apr 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Decitabine | Experimental | Drug: DecitabineSubjects 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (24 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DecitabineDemethylating agents | Drug | Decitabine |
Primary outcome
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
9 secondary outcomes
Incidence of drug-related adverse events
Time frame: Within 52 weeks
Proportion of subjects achieving a hematological response (any of the platelet response, erythroid response, and neutrophil response)
Time frame: Within 52 weeks
Proportion of subjects with transfusion independence or decreased transfusion requirement
Time frame: Within 52 weeks
Absolute changes in blood cell count
Time frame: Within 52 weeks
The utility score of EQ-5D-5L questionnaire
Time frame: Baseline, 20 weeks, 52 weeks
Time from the first decitabine to hematologic response
Time frame: Within 20 weeks
Duration of hematologic response
Time frame: Within 52 weeks
Proportion of relapse
Time frame: Within 52 weeks
Proportion of clonal evolution
Time frame: Within 52 weeks
Results and safety
The study was stopped early (reason given: Not achieve the desired effect). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Decitabine in people with Refractory Aplastic Anemia, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 11 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Not achieve the desired effect). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2026.