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NCT Number: NCT07558434

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

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)

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Key information

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age ≥ 18 years old;
  • 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;
  • 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;

Exclusion criteria

  • 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.

Treatment and study plan

Cyclosporine (CsA)

Drug

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

thrombopoietin receptor agonist (TPO-RA)

Drug

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)

Romiplostim N01

Drug

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

Primary outcomes

  1. Overall response rate (ORR)

    Time frame: 6-month

    ORR=CRR+PRR

Secondary outcomes

  1. ORR

    Time frame: 3-month, 12-month

    ORR=PRR+CRR

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

    Time frame: 3-month, 6-month, 12-month

    Proportion of patients who achieve red blood cell (RBC)/platelet (PLT) transfusion independence for 8 weeks or longer

  3. AE rate

    Time frame: through study completion, an average of 1 year

    proportion of patients with adverse events, according to CTCAE

Study contacts

Contact information is provided by the study sponsor or research team.

Sponsors and collaborators

Lead sponsor

Peking Union Medical College Hospital

Other

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2028
Study completion
2029
First posted
Apr 30, 2026
Registry last updated
Apr 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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