Transfusion-dependent Non-severe Aplastic AnemiaPhase 3NCT07522996
Enarodustat+CsA vs CsA in the Treatment of Newly-diagnosed TD-NSAA
A Phase 3 study testing Enarodustat and Cyclosporin (CSA) in people with Transfusion-dependent Non-severe Aplastic Anemia, run by Peking Union Medical College Hospital.
Trial summary
This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine alone in the treatment of TD-NSAA. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transfusion-dependent Non-severe Aplastic Anemia
- What is being tested?
- Enarodustat and Cyclosporin (CSA)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jun 2026to Dec 2029
- Phase
- Phase 3
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥ 18 years old;
- Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) according to the Camitta criteria; Hemoglobin<90g/L;
- Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
- With baseline liver and kidney functions <2 ULN;
- ECOG score ≤ 2;
- Signed the informed consent;
Who cannot join
- Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
- With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
- PNH clone ≥ 50%;
- Received HSCT before enrollment;
- Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs), roxadustat;
- Allergic or intolerant to enarodustat or CsA;
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- Pregnant or lactating patients;
- Severe bleeding or infection that cannot be controlled by standard treatment;
- Complicated with malignant tumors;
- Participated in other clinical trials within 3 months;
- Patients considered not suitable to participate in this study by the investigator.
About this study
Official title: Enarodustat Plus CsA Versus CsA Monotherapy in the Treatment of Newly-diagnosedTD-NSAA: a Single-center Randomized Trial
This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine alone in the treatment of TD-NSAA.
Read the detailed description
For TD-NSAA patients without HLA-matched donors, the firstline therapy is immunosuppressive therapy (IST) combined with thrombopoietin receptor agonists (TPO-RAs). However, some patients only have partial hematological responses, and their hemoglobin levels cannot be effectively increased.
Inflammatory factors such as IFN-γ, TNF-α, and IL-6 are significantly elevated in the bone marrow and peripheral blood of AA patients. Even in patients who respond effectively to IST, the proportion of CD3+ IFN+ and CD3+ TNF+ lymphocytes is still higher than that in healthy controls, and these cytokines may be involved in inhibiting the recovery of hemoglobin in patients.
HIF-PHI prevent the hydroxylation of HIF-α, thereby enabling the transcription and expression of genes related to erythropoiesis, such as those related to erythropoietin and iron transport. Roxadustat and enarodustat have been approved for the treatment of anemia in chronic kidney disease. Studies have shown that roxadustat can significantly reduce the levels of inflammatory factors such as IFN-γ, TNF-α, and IL-6 in the serum of patients with chronic kidney disease. A preliminary study has shown that roxadustat monotherapy in patients with insufficient erythroid response after IST can achieve a red blood cell response rate of 71.4%. Enarodustat has a similar mechanism to roxadustat, and in a rat model of inflammatory anemia, it was found that enarodustat can stimulate erythropoiesis by increasing iron utilization and improving inflammatory anemia. However, there are currently no studies on the use of enarodustat in AA patients.
Thus, this study aims to conduct a single-center, prospective, randomized controlled trial to compare the efficacy and safety of CsA+enarodustat with CsA monotherapy in newly diagnosed TD-NSAA patients.
- Being tested
- Enarodustat, Cyclosporin (CSA)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EnarodustatDrug
Enarodustat 8mg qd
Cyclosporin (CSA)Drug
CsA 3-5mg/kg/d
| Study group | Role | What they receive |
|---|---|---|
| CsA+Enarodustat | Experimental | CsA 3-5mg/kg/d，trough concentration 100-200ng/ml Enarodustat 8mg qd Receives: Drug: Enarodustat; Drug: Cyclosporin (CSA) |
| CsA monotherapy | Active comparator | CsA 3-5mg/kg/d，trough concentration 100-200ng/ml Receives: Drug: Cyclosporin (CSA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR
overall response rate (ORR) = complete response rate (CRR) + partial response rate (PRR)
Measured: 6-month
Secondary outcomes (4)
ORR
ORR = CRR + PRR
Measured: 3-month, 12-month
RBC-TI rate
Proportion of patients who achieve red blood cell transfusion independence for 8 weeks or longer
Measured: 3-month, 6-month, 12-month
hemoglobin response rate
Proportion of patients with hemoglobin response
Measured: 3-month, 6-month, 12-month
AE rate
According to CTCAE, the proportion of patients with adverse events (AEs)
Measured: through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2026
- Study startJun 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2029 (estimated)
Why it stopped: The registration of this research plan was incorrect. A new registration has been re-applied for.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transfusion-dependent Non-severe Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Bing Han, Peking Union Medical College Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07522996
- Sponsor's protocol ID
- ENA01
- Status last verified
- Jun 2026
- First submitted
- 27 Mar 2026
- Last updated
- 4 Jun 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 4 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Enarodustat Plus CsA Versus CsA Monotherapy in the Treatment of Newly-diagnosedTD-NSAA: a Single-center Randomized Trial
- ClinicalTrials.gov ID
- NCT07522996
- Acronym / sponsor ID
- ENA01
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: The registration of this research plan was incorrect. A new registration has been re-applied for.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Jun 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 13 Apr 2026 (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 study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine alone in the treatment of TD-NSAA.
Detailed description
For TD-NSAA patients without HLA-matched donors, the firstline therapy is immunosuppressive therapy (IST) combined with thrombopoietin receptor agonists (TPO-RAs). However, some patients only have partial hematological responses, and their hemoglobin levels cannot be effectively increased.
Inflammatory factors such as IFN-γ, TNF-α, and IL-6 are significantly elevated in the bone marrow and peripheral blood of AA patients. Even in patients who respond effectively to IST, the proportion of CD3+ IFN+ and CD3+ TNF+ lymphocytes is still higher than that in healthy controls, and these cytokines may be involved in inhibiting the recovery of hemoglobin in patients.
HIF-PHI prevent the hydroxylation of HIF-α, thereby enabling the transcription and expression of genes related to erythropoiesis, such as those related to erythropoietin and iron transport. Roxadustat and enarodustat have been approved for the treatment of anemia in chronic kidney disease. Studies have shown that roxadustat can significantly reduce the levels of inflammatory factors such as IFN-γ, TNF-α, and IL-6 in the serum of patients with chronic kidney disease. A preliminary study has shown that roxadustat monotherapy in patients with insufficient erythroid response after IST can achieve a red blood cell response rate of 71.4%. Enarodustat has a similar mechanism to roxadustat, and in a rat model of inflammatory anemia, it was found that enarodustat can stimulate erythropoiesis by increasing iron utilization and improving inflammatory anemia. However, there are currently no studies on the use of enarodustat in AA patients.
Thus, this study aims to conduct a single-center, prospective, randomized controlled trial to compare the efficacy and safety of CsA+enarodustat with CsA monotherapy in newly diagnosed TD-NSAA patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CsA+Enarodustat | Experimental | Drug: Enarodustat, Drug: Cyclosporin (CSA)CsA 3-5mg/kg/d，trough concentration 100-200ng/ml Enarodustat 8mg qd |
| CsA monotherapy | Active comparator | Drug: Cyclosporin (CSA)CsA 3-5mg/kg/d，trough concentration 100-200ng/ml |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age ≥ 18 years old;
- Diagnosed with aplastic anemia (AA) through routine blood tests, bone marrow puncture, bone marrow biopsy, and exclusion tests, and determined as transfusion-dependent non-severe aplastic anemia (TD-NSAA) according to the Camitta criteria; Hemoglobin<90g/L;
- Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
- With baseline liver and kidney functions <2 ULN;
- ECOG score ≤ 2;
Key exclusion criteria (11 listed)
- Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;
- With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;
- PNH clone ≥ 50%;
- Received HSCT before enrollment;
- Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs), roxadustat;
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 |
|---|---|---|
| EnarodustatEnarodustat 8mg qd | Drug | CsA+Enarodustat |
| Cyclosporin (CSA)CsA 3-5mg/kg/d | Drug | CsA monotherapy, CsA+Enarodustat |
Primary outcome
ORR
Time frame: 6-month
overall response rate (ORR) = complete response rate (CRR) + partial response rate (PRR)
4 secondary outcomes
ORR
Time frame: 3-month, 12-month
RBC-TI rate
Time frame: 3-month, 6-month, 12-month
hemoglobin response rate
Time frame: 3-month, 6-month, 12-month
AE rate
Time frame: through study completion, an average of 1 year
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Jun 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.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Enarodustat and Cyclosporin (CSA) in people with Transfusion-dependent Non-severe Aplastic Anemia, run by Peking Union Medical College Hospital.
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 6 inclusion and 11 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: withdrawn, as of the sponsor's last update on 4 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 withdrawn before enrolling participants, so there are no results.
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?
ORR (time frame: 6-month). 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 4 Jun 2026.