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A Prospective, Multicenter Study on the Efficacy and Safety of Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia (CIT) in Children, Adolescents, and Young Adults (CAYA) With Hematological and Solid Tumors

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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 (≥100×10⁹/L or an increase of ≥30×10⁹/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 µg/kg). * Have their romiplostim dose adjusted weekly based on platelet counts (increase by 1-2 µg/kg if platelets \<99×10⁹/L, maximum dose 10 µg/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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 24
Healthy Volunteers: f
View:

• Voluntary participation with signed informed consent.

• Aged 6-24 years, any gender.

• ECOG performance status 0-2.

• Histologically/cytologically confirmed non-myeloid malignancy requiring high-intensity chemotherapy with ≥1 myelosuppressive agent.

• Patients with potential curative opportunity eligible for standard therapy.

• Chemotherapy-induced thrombocytopenia (platelets \<75×10⁹/L).

• Anticipated survival ≥8 months.

• Planned ≥2 additional chemotherapy cycles (21-/28-day cycles).

• Laboratory parameters meeting:

‣ Renal function: Cr ≤1.5×ULN; Ccr ≥55 mL/min.

⁃ Hepatic function:

• Total bilirubin ≤1.5×ULN; ALT/AST ≤3×ULN;

∙ For liver metastasis/cholangiocarcinoma: bilirubin ≤3×ULN, transaminases ≤5×ULN.

⁃ No participation in other drug trials within 4 weeks.

⁃ Good compliance with efficacy/safety follow-up per protocol.

⁃ Absence of severe complications (e.g., active GI bleeding/perforation, jaundice, obstruction, non-cancer fever \>38°C).

⁃ Ability to comprehend and sign informed consent.

Locations
Other Locations
China
Cancer Hospital, Chinese Academy of Medical Sciences
RECRUITING
Beijing
Contact Information
Primary
Sidan Li
lisidan2006@126.com
+86-0316-5917421
Time Frame
Start Date: 2025-12-25
Estimated Completion Date: 2026-09-01
Participants
Target number of participants: 50
Treatments
Experimental: Romiplostim N01
Participants receive weekly subcutaneous injections of romiplostim N01 (250 μg/vial; Qilu Pharmaceutical) starting at 2 μg/kg.
Related Therapeutic Areas
Sponsors
Leads: Cancer Institute and Hospital, Chinese Academy of Medical Sciences

This content was sourced from clinicaltrials.gov

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