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

NCT07523009 | Not Yet Recruiting Phase 2

• 1 other identifier: LP-N01
• Study Type: interventional
• Target: 43
• Locations: 0 countries
• Sites: N/A

Brief Summary

This study aimed to explore the efficacy and safety of cyclosporine (CsA) combined with eltrombopag (EPAG)/hetrombopag (HPAG) and romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe aplastic anemia (SAA)

Conditions

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

Outcome Measures

Primary Outcomes (1)

• Overall response rate (ORR)

  ORR=CRR+PRR

  6-month

Secondary Outcomes (3)

• ORR

  3-month, 12-month

• red blood cell (RBC)/platelet (PLT) transfusion independent rate

  3-month, 6-month, 12-month

• AE rate

  through study completion, an average of 1 year

Study Arms (1)

CsA+EPAG/HPAG+Romiplostim N01

EXPERIMENTAL

CsA 3-5mg/kg/d, trough concentration 100-200ng/ml Eltrombopag 50mg/d, increased by 25mg every two weeks Hetrombopag 7.5mg/d, increased by 2.5mg every two weeks Romiplostim N01 20µg/kg subcutaneously, once a week

Drug: Cyclosporine (CsA); Drug: Thrombopoietin Receptor Agonist; Drug: Romiplostim N01

Interventions

Cyclosporine (CsA) DRUG

CsA 3-5mg/kg/d

CsA+EPAG/HPAG+Romiplostim N01

Thrombopoietin Receptor Agonist DRUG

Eltrombopag: initial dose 50mg/d, maximum dose 150mg/d hetrombopag 7.5mg/d, maximum dose 15mg/d

CsA+EPAG/HPAG+Romiplostim N01

Romiplostim N01 DRUG

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

CsA+EPAG/HPAG+Romiplostim N01

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);
• Not suitable for ATG, due to reasons such as age, complications, and the patient's own wishes;
• 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);
• Allergic or intolerant to romiplostim N01, eltrombopag, hetrombopag, or CsA;
• Pregnant or lactating patients;
• Severe bleeding or infection that cannot be controlled by standard treatment;
• History of arterial or venous thrombosis;
• 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

Conditions

Anemia, Aplastic

Interventions

Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

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

Central Study Contacts

Bing Han, Doctor

CONTACT

+86 13601059938; hanbing_li@sina.com.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: March 27, 2026
• First Posted: April 13, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share