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

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

Romiplostim N01 will be tried to raise platelet counts in children and young adults (ages 6–24) who develop low platelets after chemotherapy for solid tumors or blood cancers.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 6 Years to 24 Years
• Sex: All
• Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences (Academic / other)
• Drugs / interventions: chemotherapy
• Locations: 1 site (Beijing)
• Trial ID: NCT07043894 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Ideal candidates are patients aged 6–24 with confirmed non-myeloid malignancies who have chemotherapy-induced platelet counts below 75×10⁹/L, ECOG 0–2, expected survival ≥8 months, and plans for further myelosuppressive chemotherapy cycles.

Not a fit: Patients whose low platelets are not caused by chemotherapy, those with active severe bleeding, significant organ dysfunction, very limited life expectancy, or who cannot attend scheduled visits are unlikely to benefit from this intervention.

Why it matters

Potential benefit: If successful, romiplostim N01 could shorten the duration of low platelets, reduce bleeding risk, and help patients stay on their chemotherapy schedule.

How similar studies have performed: Other thrombopoietin-receptor agonists (for example hetrombopag and romiplostim formulations) have shown promise for chemotherapy-induced thrombocytopenia in adults and some pediatric reports, but romiplostim N01 in this exact CAYA population remains specifically being tested.

Eligibility criteria

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 this trial is running

Beijing

• 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

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