Actively Recruiting
Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients
Led by Cancer Institute and Hospital, Chinese Academy of Medical Sciences · Updated on 2026-05-07
50
Participants Needed
1
Research Sites
35 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Voluntary participation with signed informed consent.
- Age between 6 and 24 years, any gender.
- ECOG performance status of 0 to 2.
- Confirmed non-myeloid cancer requiring intensive chemotherapy with at least one myelosuppressive drug.
- Eligible for standard therapy with potential for cure.
- Chemotherapy-induced thrombocytopenia with platelet count below 75 x 10⁹/L.
- Expected survival of at least 8 months.
- Planned to receive at least two additional chemotherapy cycles of 21 or 28 days.
- Adequate kidney function (creatinine ≤1.5 times upper limit; creatinine clearance ≥55 mL/min).
- Adequate liver function (total bilirubin ≤1.5 times upper limit; ALT/AST ≤3 times upper limit; higher limits allowed if liver metastasis or cholangiocarcinoma).
- No participation in other drug trials within past 4 weeks.
- Able to comply with follow-up and treatment requirements.
- No severe complications such as active gastrointestinal bleeding, jaundice, obstruction, or fever over 38°C.
- Ability to understand and sign informed consent.
You will not qualify if you...
- Blood disorders unrelated to chemotherapy-induced thrombocytopenia, including AML, ITP, MDS, MPN, and multiple myeloma.
- Thrombocytopenia from other causes within last 6 months (like chronic liver disease, enlarged spleen, infection, or bleeding).
- Known allergy to romiplostim N01 or its ingredients.
- Severe anemia (hemoglobin below 50 g/L despite treatment) or very low neutrophils (ANC below 1.0 x 10⁹/L despite treatment).
- Radiotherapy to pelvis, spine, or large fields within past 3 months.
- History of arterial or venous blood clots within 3 months.
- Serious heart conditions (severe heart failure, arrhythmias with clot risk, recent heart surgery) within 6 months.
- Use of certain blood growth factors or TPO receptor agonists within 2 weeks.
- Other risks judged by investigator that could affect safety or study results.
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Trial Site Locations
Total: 1 location
1
Cancer Hospital, Chinese Academy of Medical Sciences
Beijing, China
Actively Recruiting
Research Team
S
Sidan Li
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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