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

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 Overview

• Status: Not yet recruiting
• Conditions: Transfusion-dependent Non-severe Aplastic Anemia
• Intervention / Treatment: Drug: Enarodustat; Drug: Cyclosporine (CsA)

• Study Type: Interventional
• Enrollment (Estimated): 90
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Bing Han; Phone Number: +86 13601059938; Email: hanbing_li@sina.com.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CsA+Enarodustat; CsA 3-5mg/kg/d Enarodustat 8mg qd	Drug: Enarodustat; Enarodustat 8mg qd; Drug: Cyclosporine (CsA); CsA 3-5mg/kg/d, trough concentration 100-200ng/ml
Active Comparator: CsA; CsA 3-5mg/kg/d	Drug: Cyclosporine (CsA); CsA 3-5mg/kg/d, trough concentration 100-200ng/ml

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ORR	ORR=CRR+PRR	6-month

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029

Study Registration Dates

• First Submitted: April 16, 2026
• First Submitted That Met QC Criteria: April 16, 2026
• First Posted (Actual): April 23, 2026

Study Record Updates

• Last Update Posted (Actual): April 23, 2026
• Last Update Submitted That Met QC Criteria: April 16, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Peptides; Amino Acids, Peptides, and Proteins; Polycyclic Compounds; Macrocyclic Compounds; Peptides, Cyclic; Cyclosporins; Cyclosporine; enarodustat
• Other Study ID Numbers: K10247

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No