GALOP II Protocol for the Treatment of Unilateral Retinoblastoma
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 84
- 试验地点
- 2
- 主要终点
- Evaluation of number of patients experiencing acute, chronic and fatal toxicities
研究概览
简要总结
This protocol provides guidelines for the management of non-metastatic unilateral retinoblastoma and introduces an innovative adjuvant therapy for higher risk patients based upon the results of the Grupo de America Latina de Oncologia Pediatrica (GALOP) I study. Conservative therapy will be not protocolized.
详细描述
Patients with non-metastatic retinoblastoma undergoing enucleation will be staged using the American Joint Committee on Cancer (AJCC), version 8, Tumor, Node, Metastasis (TNM)- H system and the International Retinoblastoma Staging System (IRSS). IRSS stage I patients recognized as higher risk will be assigned for adjuvant therapy. Those with standard risk will not receive adjuvant therapy after enucleation. Higher risk patients are defined as those with pathological retrolaminar optic nerve invasion and-or any degree of scleral invasion (pT3b, pT3c, pT3d). Based on the results of the GALOP I protocol, they will receive a reduced dose adjuvant chemotherapy regimen with 3 cycles of alternating cyclophosphamide, vincristine and idarubicin alternating with another 3 cycles of carboplatin and etoposide. Six doses of intrathecal topotecan will be given. Patients presenting with severe buphthalmia (cT3c-cT3e) will receive neo-adjuvant therapy with the same intensive regimen but including a higher dose of carboplatin as per GALOP I protocol plus intrathecal topotecan and secondary enucleation followed by adjuvant chemotherapy for a total of 8 cycles. Stage II patients (pT4) will receive the same adjuvant regimen plus orbital radiotherapy (45 cGy).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 0 Years 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Histological diagnosis of retinoblastoma confirmed at participating institutions
- •Ophthalmological diagnosis of unilateral retinoblastoma in patients undergoing conservative therapy.
- •No prior therapy for retinoblastoma
- •Lansky Performance Scale greater or equal to 50
- •Normal organ function in those patients assigned for chemotherapy
- •Signed informed consent
排除标准
- •Patients with unilateral retinoblastoma and germline mutations of the Rb1 gene or family history for retinoblastoma
研究组 & 干预措施
Higher Risk Patients
Patients with IRSS stage I, pT3b, pT3c, pT3d will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan
干预措施: Combination Chemotherapy plus Intrathecal Topotecan (Combination Product)
Patients with buphthalmus
Patients with buphthalmus (cT3c, cT3e) will receive 2 cycles of neo-adjuvant chemotherapy plus 6 doses of intrathecal topotecan followed by secondary enucleation and 6 cycles of adjuvant chemotherapy.
干预措施: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan (Combination Product)
Stage II Patients
Patients with Stage II (pT4) will receive 6 cycles of adjuvant chemotherapy plus 6 doses of intrathecal topotecan and orbital radiotherapy
干预措施: Higher Dose Combination Chemotherapy plus Intrathecal Topotecan and Orbital Radiotherapy (Combination Product)
结局指标
主要结局
Evaluation of number of patients experiencing acute, chronic and fatal toxicities
时间窗: 5 years
Number of participants experiencing treatment-related adverse effects and mortality as measured by CTCAE v4.0
Rate of extraocular relapses
时间窗: 3 years
Number of participants experiencing extraocular relapse
次要结局
未报告次要终点
研究者
Pedro Zubizarreta
MD, Head Hematology and Oncology Department
Hospital JP Garrahan