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

Launched by INSTITUTE OF HEMATOLOGY & BLOOD DISEASES HOSPITAL, CHINA · Sep 23, 2024

Keywords

ClinConnect Summary

This clinical trial is exploring a new treatment approach for severe aplastic anemia (SAA), a serious condition where the body doesn't produce enough blood cells. Researchers are studying a combination of medications, including Romiplostim, which helps increase blood cell production, along with immunosuppressive therapy. The trial will involve 48 patients who are newly diagnosed with SAA and are not eligible for certain types of stem cell transplants.

To participate, individuals must be at least 12 years old and have a confirmed diagnosis of severe aplastic anemia. Participants should not have received specific prior treatments and must have no serious health issues that could interfere with the study. Throughout the trial, participants will receive the study drugs and be monitored for their safety and how well the treatment works. This study aims to provide valuable information about a potential new first-line treatment for this challenging condition.

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Eligibility criteria

• 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