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

Conditions

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

Eligibility Criteria

Sex

ALL

Age

3 Years - 18 Years

Healthy Volunteers

Not accepted

Interventions

• Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib — DRUG
  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.

Study Details

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.

Key Dates

Start date

Mar 1, 2026

Status verified

Feb 2026

Primary completion

Dec 1, 2027

Completion

Jun 6, 2032

Study Design

Enrollment

33 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• 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.

Primary Outcome Measure

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) ]