Interventional, Single-arm, Open-label Open-label, Phase II Trial to Evaluate the Role of Anthracycline Infusion After Radio Therapy (RT) in Pediatric and Young Adults With Glioblastoma (pGBM).
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- Iacopo Sardi
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Percentage of Withdrawal from the study rate
研究概览
简要总结
Glioblastoma (GBM) and diffuse intrinsic bridge gliomas (DIPG) only the most aggressive forms of cancer, and their prognosis remains bleak. Currently, the standard of treatment is TMZ concomitant with radiotherapy, and, at the end of combined treatment, as adjuvant therapy. In vitro and in vivo experimental studies have suggested that anthracyclines are effective antineoplastics for the treatment of gliomas. In patients with solid tumors treated with anthracyclines, continuous infusion administration compared with bolus administration has been shown to provide a better safety profile especially with regard to cardiotoxicity. Based on this evidence, this study aims to evaluate the safety and antitumor activity of combined treatment with Dox, WBRT (whole body radiotherapy), and TMZ in pediatric and young adult patients affected by GMB
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 30 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with histological-molecular diagnosis according to WHO 2016 classification: IDH-wildtype glioblastoma (9440/3), giant cell glioblastoma (9441/3), gliosarcoma (9442/3), epithelioid glioblastoma (9440/3), IDH-mutated glioblastoma (9445/3), glioblastoma NOS (9440/3), diffuse astrocytoma (9400/3), diffuse midline glioma H3 K27M mutated, including multifocal, metastatic or gliomatosis cerebri pictures of first diagnosis Not previously treated (with chemo and radiotherapy) or treated only surgically (total, near partial, partial, biopsy).
- •Males and females between the ages of 3 and 30 years old
- •Life expectancy ≥ 12 months
- •karnofsky/Lansky ≥ 80 %
- •Adequate hematologic function: Absolute leukocyte count ≥ 2.0 x 109/l, Hemoglobin ≥ 10 g/dl, Platelet count ≥ 50 x 109/l
- •Adequate liver function: Total bilirubin ≤ 2.5 x ULN, ALT/AST ≤ 5.0 x ULN
- •Adequate renal function:Serum creatinine ≤ 1.5 x ULN
- •Written informed consent from the patient, parents or legal guardians
- •Patient's willingness during treatment and ability to comply with the protocol
排除标准
- •Evidence of any other serious disease or condition that is a contraindication to study therapy (e.g. severe mental retardation, severe cerebral palsy, severe syndromes congenital syndromes, heart disease)
- •Performance of a course of 1st-line chemotherapy at the same time as study initiation
- •Concurrent participation in other research projects
- •Pregnancy or lactation status
- •Use of inappropriate contraceptive methods
研究组 & 干预措施
pGBM patients therapy
- Whole therapy radiation therapy
- Temozolomide concomitant
- After 1 month adjuvant Temozolomide,
- After 3 months Doxorubicine
- adjuvant Temozolomide
干预措施: Radiotherapy, Temozolomide, Doxorubicin (Drug)
结局指标
主要结局
Percentage of Withdrawal from the study rate
时间窗: through study completion, an average of 1 year
Percentage of subjects with SAE leading to withdrawal from the study
Percentage of SAEs
时间窗: through study completion, an average of 1 year
Percentage of SAEs
Evaluation prolonged Dox
时间窗: through study completion, an average of 1 year
Time to early withdrawal from experimental treatment with Dox
Early discontinuation of dox treatment rate
时间窗: through study completion, an average of 1 year
Proportion of early discontinuation of experimental treatment with Dox
Mortality rate
时间窗: through study completion, an average of 1 year
Mortality from adverse events
次要结局
- Event-free survival (EFS), disease progression (PFS), and overall survival (OS)(through study completion, an average of 1 year)
研究者
Iacopo Sardi
Princiapl Investigator
Meyer Children's Hospital IRCCS