Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA

Efficacy and Safety of Enarodustat Combined With Cyclosporine Versus Cyclosporine Monotherapy in the Treatment of Newly Diagnosed TD-NSAA

Status

Not yet recruiting

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07548671

Enrollment

Registered

2026-04-23

Start date

2026-04-01

Completion date

2029-12-01

Last updated

2026-04-23

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Transfusion-dependent Non-severe Aplastic Anemia

Brief 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).

Interventions

DRUGEnarodustat

Enarodustat 8mg qd

DRUGCyclosporine (CsA)

CsA 3-5mg/kg/d, trough concentration 100-200ng/ml

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Age ≥ 18 years old; 2. 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; 3. Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT); 4. With baseline liver and kidney functions \<2 ULN; 5. ECOG score ≤ 2; 6. Signed the informed consent;

Exclusion criteria

1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.; 2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics; 3. PNH clone ≥ 50%; 4. Received HSCT before enrollment; 5. Previously used immunosuppressive treatments such as ATG, CsA, TPO receptor agonists (TPO-RAs), roxadustat; 6. Allergic or intolerant to enarodustat or CsA; 7. Pregnant or lactating patients; 8. Severe bleeding or infection that cannot be controlled by standard treatment; 9. Complicated with malignant tumors; 10. Participated in other clinical trials within 3 months; 11. Patients considered not suitable to participate in this study by the investigator.

Design outcomes

Primary

Measure	Time frame	Description
ORR	6-month	ORR=CRR+PRR

Secondary

Measure	Time frame	Description
ORR	3-month, 12-month	ORR=CRR+PRR
RBC-TI rate	3-month, 6-month, 12-month	Proportion of patients who achieve red blood cell transfusion independence for 8 weeks or longer
hemoglobin response rate	3-month, 6-month, 12-month	Proportion of patients with hemoglobin response
AE rate	through study completion, an average of 1 year	According to CTCAE, the proportion of patients with adverse events (AEs)

Contacts

CONTACTBing Han

hanbing_li@sina.com.cn+86 13601059938

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 24, 2026