Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients

Launched by CANCER INSTITUTE AND HOSPITAL, CHINESE ACADEMY OF MEDICAL SCIENCES · Jun 21, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a medicine called romiplostim N01 to help children and young adults (ages 6 to 24) with cancer who are experiencing low platelet counts due to chemotherapy. Platelets are blood cells that help stop bleeding, and chemotherapy can sometimes lower their numbers, which can lead to bleeding problems. The trial aims to see how well romiplostim N01 can increase platelet counts within three weeks and to check if the medicine is safe for these young patients.

If eligible, participants will receive weekly injections of romiplostim under the skin, with the dose adjusted based on their platelet levels. They will have regular blood tests and health check-ups to monitor their response and watch for side effects, especially bleeding or blood clots. To join, patients need to have certain types of cancer treated with strong chemotherapy, have low platelet counts caused by chemotherapy, and be expected to continue chemotherapy for at least two more cycles. The study does not compare romiplostim to other treatments but looks at how patients respond compared to past results. This trial is not yet recruiting participants.

Gender

ALL

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.