Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients

NCT07043894 | Recruiting Phase 2

• 1 other identifier: NCCH0011
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Solid Tumors; Hematological Tumor; Children; Adolescent; Chemotherapy Induced Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• Proportion of patients achieving platelet count recovery to ≥100×10⁹/L or an increase of ≥30×10⁹/L from baseline

  3 weeks

Secondary Outcomes (11)

• Median time to platelets ≥100×10⁹/L or ≥30×10⁹/L increase from baseline

  3 weeks

• Nadir platelet count

  3 weeks

• Mean time to platelets ≥100×10⁹/L or ≥30×10⁹/L increase

  3 weeks

• Platelet increase from baseline

  3 weeks

• Median time to peak platelets

  3 weeks

Study Arms (1)

Romiplostim N01

EXPERIMENTAL

Participants receive weekly subcutaneous injections of romiplostim N01 (250 μg/vial; Qilu Pharmaceutical) starting at 2 μg/kg.

Drug: Romiplostim N01

Interventions

Romiplostim N01 DRUG

Participants receive weekly subcutaneous injections of romiplostim N01 (250 μg/vial; Qilu Pharmaceutical) starting at 2 μg/kg. Doses are adjusted weekly based on platelet counts: increased by 2 μg/kg if platelets are \<50 × 10⁹/L, by 1 μg/kg if 50-99 × 10⁹/L, up to a maximum of 10 μg/kg. Treatment continues for up to 2 weeks but stops early if platelets reach ≥100 × 10⁹/L or increase by ≥30 × 10⁹/L from baseline.

Romiplostim N01

Eligibility Criteria

• Age: 6 Years - 24 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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.

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.

Sponsors & Collaborators

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

Study Sites (1)

Cancer Hospital, Chinese Academy of Medical Sciences

Beijing, China

RECRUITING

MeSH Terms

Conditions

Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Sidan Li

  Cancer Institute and Hospital, Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Sidan Li

CONTACT

+86-0316-5917421; lisidan2006@126.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof

Model Details: This study is a multicenter, open-label, single-arm trial. It plans to enroll 50 pediatric and adolescent patients with hematologic or solid tumors who are receiving high-intensity chemotherapy regimens. The study aims to evaluate the efficacy and safety of lusutrombopag N01 in treating chemotherapy-induced thrombocytopenia in this patient population. Patient data from all participating centers will be collected through an Electronic Data Capture (EDC) system. After signing the informed consent form, each subject will be assigned a unique screening number for identification throughout the study period.

Study Record Dates

• First Submitted: June 21, 2025
• First Posted: June 29, 2025
• Study Start: December 25, 2025
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: May 7, 2026

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)