Clinical trial · Interventional
Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients
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
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (5)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Adolescent | — | UNRESOLVED | — |
| Chemotherapy Induced Thrombocytopenia | — | UNRESOLVED | — |
| Children | — | UNRESOLVED | — |
| Hematological Tumor | Hematopoietic and Lymphoid Cell Neoplasm | PROBABILISTIC | 0.70 |
| Solid Tumors | Solid Neoplasm | CURATED_BROADER | 0.80 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Romiplostim N01 | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Romiplostim N01
- description
- Participants receive weekly subcutaneous injections of romiplostim N01 (250 μg/vial; Qilu Pharmaceutical) starting at 2 μg/kg.
- interventionNames
- Drug: Romiplostim N01
Primary outcomes (1)
- measure
- Proportion of patients achieving platelet count recovery to ≥100×10⁹/L or an increase of ≥30×10⁹/L from baseline
- timeFrame
- 3 weeks
Secondary outcomes (11)
- measure
- Median time to platelets ≥100×10⁹/L or ≥30×10⁹/L increase from baseline
- timeFrame
- 3 weeks
- measure
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 6 Years
- Maximum age
- 24 Years
Show eligibility criteria text
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.References
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