CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA

Cyclosporine + Eltrombopag/Hetrombopag + Romiplostim N01 in the Treatment of Newly-diagnosed Transfusion-dependent Non-severe Aplastic Anemia/ Severe Aplastic Anemia

This study aimed to explore the efficacy and safety of cyclosporine (CsA)

+eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)

Study Overview

• Status: Not yet recruiting
• Conditions: Severe Aplastic Anemia (SAA); Transfusion-dependent Non-severe Aplastic Anemia
• Intervention / Treatment: Drug: Cyclosporine (CsA); Drug: thrombopoietin receptor agonist (TPO-RA); Drug: Romiplostim N01

• Study Type: Interventional
• Enrollment (Estimated): 43
• Phase: Phase 2

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) or severe aplastic anemia (SAA) according to the Camitta criteria; Platelet < 30×10^9/L;
3. Had no HLA-matched donors or was not suitable for first-line allogeneic hematopoietic stem cell transplantation (HSCT);
4. Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;
5. With baseline liver and kidney functions <2 ULN;
6. ECOG score ≤ 2;
7. 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);
6. Allergic or intolerant to romiplostim N01, eltrombopag, hetrombopag, or CsA;
7. Pregnant or lactating patients;
8. Severe bleeding or infection that cannot be controlled by standard treatment;
9. History of arterial or venous thrombosis;
10. Complicated with malignant tumors;
11. Participated in other clinical trials within 3 months;
12. 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: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CsA+EPAG/HPAG+Romiplostim N01; CsA 3-5mg/kg/d EPAG 50mg/d or HPAG 7.5mg/d Romiplostim N01 20µg/kg, subcutaneously, once a week	Drug: Cyclosporine (CsA); CsA 3-5mg/kg/d, trough concentration 100-200ng/ml; Drug: thrombopoietin receptor agonist (TPO-RA); Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks; Other Names: Eltrombopag (EPAG); Hetrombopag (HPAG); Drug: Romiplostim N01; Romiplostim N01 20µg/kg, subcutaneously, once a week

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate (ORR)	ORR=CRR+PRR	6-month

Secondary Outcome Measures

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

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 23, 2026
• First Submitted That Met QC Criteria: April 23, 2026
• First Posted (Actual): April 30, 2026

Study Record Updates

• Last Update Posted (Actual): April 30, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Failure Disorders; Hematologic Diseases; Bone Marrow Diseases; Anemia; Hemic and Lymphatic Diseases; Anemia, Aplastic; Peptides; Amino Acids, Peptides, and Proteins; Polycyclic Compounds; Macrocyclic Compounds; Peptides, Cyclic; Cyclosporins; Cyclosporine; eltrombopag; hetrombopag
• Other Study ID Numbers: K10230

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