Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients

• Status: NOT_YET_RECRUITING
• Trial end date: 2026-09-01

Summary

The goal of this clinical trial is to evaluate the efficacy and safety of romiplostim N01 in treating chemotherapy-induced thrombocytopenia (CIT) in children and adolescents/young adults (CAYA; aged 6-24 years) with hematologic malignancies or solid tumors. The main questions it aims to answer are:

• What proportion of participants achieve platelet count recovery (10010/L or an increase of 3010/L from baseline) within 3 weeks of romiplostim N01 treatment?
• What is the safety profile of romiplostim N01 in this population, including the incidence and severity of adverse events (especially bleeding and thrombosis)? This is a single-arm study (no comparison group). Researchers will assess the outcomes against predefined efficacy thresholds and historical data (e.g., a 60.7% response rate reported for another TPO-RA, hetrombopag).

Participants will:

• Receive weekly subcutaneous injections of romiplostim N01 (starting dose: 2 g/kg).
• Have their romiplostim dose adjusted weekly based on platelet counts (increase by 1-2 g/kg if platelets \<9910/L, maximum dose 10 g/kg, stop when target recovery is met).
• Undergo frequent monitoring, including blood tests (especially platelet counts), vital signs, physical exams, and assessment for adverse events and bleeding throughout the treatment and follow-up period.

• Phase: PHASE2

Details

• Lead Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences