Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA

NCT07548671 | Not Yet Recruiting Phase 3

• 1 other identifier: K10247
• Study Type: interventional
• Target: 90
• Locations: 0 countries
• Sites: N/A

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).

Conditions

Transfusion-dependent Non-severe Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• ORR

  ORR=CRR+PRR

  6-month

Secondary Outcomes (4)

• ORR

  3-month, 12-month

• RBC-TI rate

  3-month, 6-month, 12-month

• hemoglobin response rate

  3-month, 6-month, 12-month

• AE rate

  through study completion, an average of 1 year

Study Arms (2)

CsA+Enarodustat

EXPERIMENTAL

CsA 3-5mg/kg/d Enarodustat 8mg qd

Drug: Enarodustat; Drug: Cyclosporine (CsA)

CsA

ACTIVE COMPARATOR

CsA 3-5mg/kg/d

Drug: Cyclosporine (CsA)

Interventions

Enarodustat DRUG

Enarodustat 8mg qd

CsA+Enarodustat

Cyclosporine (CsA) DRUG

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

CsA; CsA+Enarodustat

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years old;
• 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;

You may not qualify if:

• 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;
• 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.

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

MeSH Terms

Interventions

enarodustat; Cyclosporine

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Central Study Contacts

Bing Han

CONTACT

+86 13601059938; hanbing_li@sina.com.cn

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: April 16, 2026
• First Posted: April 23, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: April 23, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share