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
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 33
- 主要终点
- Objective Response Rate (ORR)
研究概览
简要总结
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.
- 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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
- •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).
排除标准
- •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.
研究组 & 干预措施
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.
干预措施: Liposomal Irinotecan, Vincristine, Temozolomide, and Optional Anlotinib (Drug)
结局指标
主要结局
Objective Response Rate (ORR)
时间窗: At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months)
次要结局
- Disease Control Rate (DCR)(At Week 6, Week 12, Week 18, and every 6 weeks thereafter until treatment discontinuation (up to approximately 6 months))
- Overall Survival (OS)(From treatment initiation until death, up to 5 years of follow-up.)
- Progression-Free Survival (PFS)(From treatment initiation until progression/death, up to 5 years of follow-up.)
- Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)(From first dose of study treatment until 30 days after the last dose (up to approximately 6 months))