Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients

Recruiting now · Phase 2

Conditions studied: Solid Tumors, Hematological Tumor, Children, Adolescent, Chemotherapy Induced Thrombocytopenia

In brief

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.

Key facts

Study ID: NCT07043894
Run by: Cancer Institute and Hospital, Chinese Academy of Medical Sciences
People needed: 50
Starts: 2025-12-25
Expected to finish: 2026-09-01
Last updated by the study team: 2026-05-07

Who can join

Age: 6 and older, up to 24. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

Hematologic disorders (non-CIT etiology): AML, ITP, MDS, MPN, multiple myeloma, etc.
Non-CIT thrombocytopenia within 6 months (e.g., chronic liver disease, hypersplenism, infection, hemorrhage).
Known hypersensitivity to romiplostim N01 or excipients (cellulose-lactose, L-HPC, magnesium stearate, film coating).
Refractory cytopenias:
Hemoglobin <50 g/L despite RBC/EPO;
ANC <1.0×10⁹/L despite G-CSF.
Pelvic/spinal/large-field radiotherapy within 3 months.
Arterial/venous thrombosis within 3 months.
Severe cardiovascular disease (NYHA Class III-IV, arrhythmia with thromboembolic risk, post-CABG/stent) within 6 months.
Use of rhTPO, rhIL-11, or TPO-RAs (eltrombopag/avatrombopag/hetrombopag) within 2 weeks.
Investigator-assessed risks compromising safety/efficacy evaluation.

Where it is running

Cancer Hospital, Chinese Academy of Medical Sciences — Beijing, China (enrolling)

Full record on ClinicalTrials.gov

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