NCT07043894RecruitingPhase 2
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 · Last updated 7 May 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 50 (estimated)
- First submitted
- 21 Jun 2025
- Start date
- 25 Dec 2025
- Primary completion date
- 1 Sept 2026
- Lead sponsor
- Cancer Institute and Hospital, Chinese Academy of Medical Sciences
- Diseases
- Childhood cancers
- NCT number
- NCT07043894
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 50
- From 25 Dec 2025 to the primary completion date, 1 Sept 2026
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Pelvic/spinal/large-field radiotherapy within 3 months.
Other illness
- 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).
- Arterial/venous thrombosis within 3 months.
- Severe cardiovascular disease (NYHA Class III-IV, arrhythmia with thromboembolic risk, post-CABG/stent) within 6 months.
Lab values
- 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.
Other
- Use of rhTPO, rhIL-11, or TPO-RAs (eltrombopag/avatrombopag/hetrombopag) within 2 weeks.
- Investigator-assessed risks compromising safety/efficacy evaluation.
Who the study is looking for
- 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.
- Absence of severe complications (e.g., active GI bleeding/perforation, jaundice, obstruction, non-cancer fever \>38°C).
- Ability to comprehend and sign informed consent.
Age in the registry: 6 years to 24 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Cancer Hospital, Chinese Academy of Medical Sciences Coordinator of this site? Confirm its status | Beijing | China | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.