Liposomal Irinotecan, Vincristine, Temozolomide, and Anlotinib for R/R Pediatric Solid Tumors

March 2, 2026 updated by: Tianjin Medical University Cancer Institute and Hospital

A Prospective, Single-Center, Single-Arm Study to Evaluate the Efficacy and Safety of Liposomal Irinotecan, Vincristine, and Temozolomide With or Without Anlotinib in Children and Adolescents With Relapsed or Refractory Malignant Solid Tumors

This is a prospective, single-center, single-arm interventional trial conducted at Tianjin Medical University Cancer Institute and Hospital. It evaluates the efficacy and safety of a combination regimen (liposomal irinotecan + vincristine + temozolomide ± anlotinib) in children with relapsed or refractory malignant solid tumors, with the goal of optimizing regimen-related adverse reactions and exploring appropriate administration strategies.

  1. Study Objectives Primary Objective: Assess the objective response rate (ORR) of the combination regimen in the study population.

    Secondary Objectives: Evaluate survival outcomes (progression-free survival [PFS], overall survival [OS]) and the incidence of adverse events (per NCI CTCAE v5.0).

  2. Eligibility Criteria Inclusion Criteria Aged 3-18 years (inclusive) at consent; Pathologically confirmed relapsed or refractory malignant solid tumors (e.g., neuroblastoma, rhabdomyosarcoma); At least one measurable lesion (per RECIST v1.1); Adequate functional status and organ function (per institutional standards); Written informed consent from subject/legal guardian. Exclusion Criteria Hypersensitivity to study drugs; Active uncontrolled infection or severe comorbidities; Concurrent participation in other interventional trials; Conditions precluding study participation (per investigator judgment).

  3. Intervention Procedures

    Eligible subjects are assigned to short-course or long-course subgroups based on clinical status:

    Short-course: Liposomal irinotecan (Day 1), vincristine (Day 1), temozolomide (Days 1-5), ± weight-based anlotinib (Days 1-14); Long-course: Liposomal irinotecan (Days 1/8/15), plus the same doses of other drugs as the short-course subgroup.

    Each treatment cycle is 21 days. Efficacy is assessed regularly; treatment (dose/subgroup) will be adjusted per clinical condition.

  4. Outcome Assessments Primary Outcome: ORR (assessed per RECIST v1.1 at regular intervals); Secondary Outcomes: PFS, OS (long-term follow-up post-treatment), and adverse event incidence.
  5. Participant Requirements Participation is voluntary. Subjects/guardians must provide written informed consent prior to enrollment. Long-term follow-up will be conducted post-treatment.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

33

Phase

  • Phase 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Aged 3 to 18 years (inclusive) at the time of informed consent signing.
  • Pathologically confirmed relapsed or refractory malignant solid tumors (including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, osteosarcoma).
  • At least one measurable lesion (as defined by RECIST v1.1) confirmed by CT/MRI within 28 days prior to enrollment.
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
  • Adequate organ function (absolute neutrophil count ≥1.5×10⁹/L; platelet count ≥100×10⁹/L; hemoglobin ≥90g/L; serum creatinine ≤1.5×upper limit of normal [ULN] for age; total bilirubin ≤1.5×ULN; alanine aminotransferase/aspartate aminotransferase ≤2.5×ULN, or ≤5×ULN if liver metastasis exists).
  • Written informed consent provided by the subject (if ≥18 years old) or legal guardian (if <18 years old).

Exclusion Criteria:

  • Known hypersensitivity to any component of the study drugs (liposomal irinotecan, vincristine, temozolomide, anlotinib).
  • Active systemic infection requiring systemic antibiotic treatment within 7 days prior to enrollment.
  • History of severe cardiovascular disease (e.g., congestive heart failure, uncontrolled arrhythmia).
  • Concurrent participation in another interventional clinical trial.
  • Pregnancy, lactation, or unwillingness to use effective contraception (for sexually active subjects).
  • Other medical conditions that, in the investigator's judgment, may affect study participation or outcome assessment.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Liposomal Irinotecan + Vincristine + Temozolomide ± Anlotinib (Short/Long Course)
Combination regimen of liposomal irinotecan, vincristine, and temozolomide (with optional anlotinib, dose based on body weight). Subjects are assigned to 2 subgroups by clinical status: short-course (liposomal irinotecan on Day 1) or long-course (liposomal irinotecan on Days 1/8/15). Other drugs follow the same dosing schedule across subgroups; treatment adjustments are made per clinical condition.
Drug: Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib
This is a combination chemotherapy regimen for children (3-18 years old) with relapsed or refractory malignant solid tumors. It includes liposomal irinotecan, vincristine, and temozolomide, with optional anlotinib (dose adjusted by body weight: 8mg/day for <35kg; 12mg/day for ≥35kg). Administration is divided into short-course (liposomal irinotecan on Day 1) and long-course (liposomal irinotecan on Days 1/8/15) subgroups; other drugs follow the same dosing schedule across subgroups. Each treatment cycle lasts 21 days, and adjustments are made based on clinical conditions.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Objective Response Rate (ORR)
Time Frame: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)

Secondary Outcome Measures

Outcome Measure
Time Frame
Disease Control Rate (DCR)
Time Frame: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
Overall Survival (OS)
Time Frame: From treatment initiation until death, up to 5 years of follow-up.
From treatment initiation until death, up to 5 years of follow-up.
Progression-Free Survival (PFS)
Time Frame: From treatment initiation until progression/death, up to 5 years of follow-up.
From treatment initiation until progression/death, up to 5 years of follow-up.
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: From first dose of study treatment until 30 days after the last dose (up to approximately 6 months)
From first dose of study treatment until 30 days after the last dose (up to approximately 6 months)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Tianjin Medical University Cancer Institute and Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 1, 2026

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

June 6, 2032

Study Registration Dates

First Submitted

February 5, 2026

First Submitted That Met QC Criteria

March 2, 2026

First Posted (Actual)

March 3, 2026

Study Record Updates

Last Update Posted (Actual)

March 3, 2026

Last Update Submitted That Met QC Criteria

March 2, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CSPC-DNY-MST-TJ01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

De-identified individual participant data (IPD) including demographic characteristics, treatment subgroup assignment (short/long course), efficacy outcomes (ORR, PFS, OS assessments per protocol), and safety data (adverse event records graded by NCI CTCAE v5.0) will be shared. All personally identifiable information (e.g., name, medical record numbers) will be removed to protect participant privacy. IPD sharing will be allowed after primary endpoint analysis (or study result publication), upon formal request by qualified researchers for legitimate medical research purposes, and subject to institutional review board (IRB) approval plus a signed data use agreement (DUA) (outlining data security/responsible use).

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Relapsed or Refractory Pediatric Malignant Solid Tumors (Including Neuroblastoma, Rhabdomyosarcoma, Ewing Sarcoma, Osteosarcoma)

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