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Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia

Recruiting
12 years and older
All
Phase 2

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Overview

This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.

Eligibility

Inclusion Criteria:

  • Age ≥ 12 years
  • Diagnosis of severe or very severe aplastic anemia
  • Patients are unconditionally or unacceptable undergoing allogeneic hematopoietic stem cell transplantation
  • ECOG performance status ≤2
  • Willing and able to comply with the requirements for this study and written informed consent.

Exclusion Criteria:

  • Received > 4 weeks of TPO-RA drug before treatment
  • Received > 4 weeks of immunosuppressive therapy before treatment
  • History of thromboembolic disease
  • Intolerance to Romiplostim N01 or cyclosporine
  • Allergy to ALG
  • Presence of uncontrolled active infection
  • Presence of uncontrolled hypertension (≥140/90mmHg), diabetes mellitus (fasting blood glucose≥ 7.0 mmol/L or random blood glucose ≥ 11.1 mmol/L)
  • Abnormal liver and kidney function: Aspartate Transaminase (AST) or Alanine Transaminase (ALT) > 3 ULN, creatinine ≥ 2.5 ULN
  • History of chemoradiotherapy for malignant solid tumors
  • History of other systemic serious illnesses
  • Females who are pregnant/lactating or need pregnancy
  • Patients considered to be ineligible for the study by the investigator for reasons other than the above

Study details
    Aplastic Anemia
    TPO Receptor Agonists

NCT06613880

Institute of Hematology & Blood Diseases Hospital, China

11 September 2025

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