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

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07558434

Enrollment

Registered

2026-04-30

Start date

2026-04-01

Completion date

2029-12-01

Last updated

2026-04-30

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

Conditions

Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia

Brief summary

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)

Interventions

DRUGCyclosporine (CsA)

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

DRUGthrombopoietin receptor agonist (TPO-RA)

Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks

DRUGRomiplostim N01

Romiplostim N01 20µg/kg, subcutaneously, once a week

Study design

Allocation

Intervention model

SINGLE_GROUP

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) 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.

Design outcomes

Primary

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

Secondary

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

Contacts

CONTACTBing Han

hanbing_li@sina.com.cn+86 13601059938

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: May 1, 2026