Romiplostim N01 for chemotherapy-related low platelets in children and young adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, open-label, single-arm Phase II trial giving weekly subcutaneous romiplostim N01 (starting 2 μg/kg) with dose adjustments based on platelet counts to children and young adults (6–24 years) who develop chemotherapy-induced thrombocytopenia. The primary endpoint is platelet recovery (≥100×10⁹/L or a ≥30×10⁹/L rise) within three weeks, and safety monitoring focuses on bleeding and thrombosis using standard grading scales. Treatment is given for up to two weeks and participants are followed across 10 visits over 36 days with serial labs and clinical assessments. Outcomes will be compared to predefined efficacy thresholds and historical response rates for other thrombopoietin-receptor agonists.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Voluntary participation with signed informed consent.
2. Aged 6-24 years, any gender.
3. ECOG performance status 0-2.
4. Histologically/cytologically confirmed non-myeloid malignancy requiring high-intensity chemotherapy with ≥1 myelosuppressive agent.
5. Patients with potential curative opportunity eligible for standard therapy.
6. Chemotherapy-induced thrombocytopenia (platelets \<75×10⁹/L).
7. Anticipated survival ≥8 months.
8. Planned ≥2 additional chemotherapy cycles (21-/28-day cycles).
9. 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.
10. No participation in other drug trials within 4 weeks.
11. Good compliance with efficacy/safety follow-up per protocol.
12. Absence of severe complications (e.g., active GI bleeding/perforation, jaundice, obstruction, non-cancer fever \>38°C).
13. Ability to comprehend and sign informed consent.

Exclusion Criteria:

1. Hematologic disorders (non-CIT etiology): AML, ITP, MDS, MPN, multiple myeloma, etc.
2. Non-CIT thrombocytopenia within 6 months (e.g., chronic liver disease, hypersplenism, infection, hemorrhage).
3. Known hypersensitivity to romiplostim N01 or excipients (cellulose-lactose, L-HPC, magnesium stearate, film coating).
4. Refractory cytopenias:

   * Hemoglobin \<50 g/L despite RBC/EPO;
   * ANC \<1.0×10⁹/L despite G-CSF.
5. Pelvic/spinal/large-field radiotherapy within 3 months.
6. Arterial/venous thrombosis within 3 months.
7. Severe cardiovascular disease (NYHA Class III-IV, arrhythmia with thromboembolic risk, post-CABG/stent) within 6 months.
8. Use of rhTPO, rhIL-11, or TPO-RAs (eltrombopag/avatrombopag/hetrombopag) within 2 weeks.
9. Investigator-assessed risks compromising safety/efficacy evaluation.

Where it's running

• Cancer Hospital, Chinese Academy of Medical Sciences
  Beijing, China Recruiting

Study contacts

• Principal investigator: Sidan Li — Cancer Institute and Hospital, Chinese Academy of Medical Sciences
• Study coordinator: Sidan Li
• Email: lisidan2006@126.com
• Phone: +86-0316-5917421

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.