Transfusion-dependent Non-severe Aplastic AnemiaPhase 3NCT07548671
Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA
A Phase 3 study testing Enarodustat and Cyclosporine (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 monotherapy in the treatment of newly-diagnosed tansfusion-dependent non-severe aplastic anemia (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 Cyclosporine (CsA)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- No sites listed yet
- Study dates
- Apr 2026to Dec 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Efficacy and Safety of Enarodustat Combined With Cyclosporine Versus Cyclosporine Monotherapy in the Treatment of Newly Diagnosed TD-NSAA
This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly-diagnosed tansfusion-dependent non-severe aplastic anemia (TD-NSAA).
- Being tested
- Enarodustat, Cyclosporine (CsA)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EnarodustatDrug
Enarodustat 8mg qd
Cyclosporine (CsA)Drug
CsA 3-5mg/kg/d, trough concentration 100-200ng/ml
| Study group | Role | What they receive |
|---|---|---|
| CsA+Enarodustat | Experimental | CsA 3-5mg/kg/d Enarodustat 8mg qd Receives: Drug: Enarodustat; Drug: Cyclosporine (CsA) |
| CsA | Active comparator | CsA 3-5mg/kg/d Receives: Drug: Cyclosporine (CsA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR
ORR=CRR+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 posted23 Apr 2026
- Study startApr 2026 (estimated)
- Primary completionDec 2028 (estimated)
- Study completionDec 2029 (estimated)
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
- 90 participants (estimated)
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
- NCT07548671
- Sponsor's protocol ID
- K10247
- Status last verified
- Apr 2026
- First submitted
- 16 Apr 2026
- Last updated
- 23 Apr 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 23 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Enarodustat Combined With Cyclosporine Versus Cyclosporine Monotherapy in the Treatment of Newly Diagnosed TD-NSAA
- ClinicalTrials.gov ID
- NCT07548671
- Acronym / sponsor ID
- K10247
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- Apr 2026 (estimated)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 23 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 monotherapy in the treatment of newly-diagnosed tansfusion-dependent non-severe aplastic anemia (TD-NSAA).
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: Cyclosporine (CsA)CsA 3-5mg/kg/d Enarodustat 8mg qd |
| CsA | Active comparator | Drug: Cyclosporine (CsA)CsA 3-5mg/kg/d |
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 |
| Cyclosporine (CsA)CsA 3-5mg/kg/d, trough concentration 100-200ng/ml | Drug | CsA, CsA+Enarodustat |
Primary outcome
ORR
Time frame: 6-month
ORR=CRR+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
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 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.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Enarodustat and Cyclosporine (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: opening soon, as of the sponsor's last update on 23 Apr 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Bing Han (+86 13601059938, hanbing_li@sina.com.cn), or a site near the patient. 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 23 Apr 2026.