Ocular Conservative Treatment for Retinoblastoma: Efficacy of the New Management Strategies and Visual Outcome - RETINO 2018
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 225
- 试验地点
- 28
- 主要终点
- Visual function
研究概览
简要总结
This protocol includes 2 independent studies. Both studies are multicenter studies, evaluating different therapeutic approaches in two different populations of patients:
- Study 1, a randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
- Study 2, a minimally invasive interventional study evaluating the results on visual function following reference treatment with intravenous (IV) chemotherapy in association with ophthalmologic or local ophthalmological treatment without IV chemotherapy.
详细描述
Primary objective of the studies:
- Study 1: To evaluate the efficacy of IAC in term of local control of the disease in patients eligible for IAC (randomized phase II study);
- Study 2: To assess the visual function, based on World Health Organization (WHO) criteria, in patients eligible for other conservative treatments (IV chemotherapy in association with local ophthalmologic trématent or local ophthalmological treatment without IV chemotherapy; this is a minimally invasive interventional study).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Months 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Overall study inclusion criteria:
- •Newly diagnosed retinoblastoma (RB).
- •Retinoblastoma with at least one eye eligible for conservative management.
- •Patients likely to be compliant with the study requirements and visits, including late follow-up.
- •Patients not previously treated with chemotherapy or radiotherapy for this or any other cancer.
- •Patients with no contraindication to the proposed treatments.
- •Informed consent signed by parents or legal representative.
- •French Social Security System coverage.
- •Study 1 inclusion criteria:
- •Children aged from 6 months to 6 years.
- •Retinoblastoma in at least one eye, deemed manageable with IAC in one side and without IV chemotherapy:
- •Unilateral Retinoblastoma classified as group B, C (if vitreous seeds < 3 mm), D or E with no invasion of the anterior segment, and without massive tumor of more than 2/3 of the eye, eligible for conservative management, or
- •Bilateral Retinoblastoma but with very unbalanced lesions between the two eyes: one eye group D or E without invasion of the anterior segment or massive tumor of more than 2/3 of the eye, eligible for IAC, and the other eye eligible for local treatment only (without IAC).
- •Study 2 inclusion criteria:
- •Retinoblastoma eligible for conservative management although not manageable with IAC:
- •Unilateral Retinoblastoma in children below 6 months of age, classified as Group A, B, C or D, with or without vitreous seeding, compatible with conservative management, or
- •Bilateral Retinoblastoma classified as group A, B, C D, or E without invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye and eligible for conservative management.
排除标准
- •Overall study non-inclusion criteria:
- •RB not eligible for conservative management :
- •Extra-ocular extension of the disease, or
- •Group E eyes with invasion of the anterior segment, and/or massive tumors of more than 2/3 of the eye.
- •Patient older than 6 years of age.
- •Patients with another associated disease contra indicating systemic chemotherapy.
- •Previously treated retinoblastoma by chemotherapy.
- •Patients already treated for another malignant disease.
- •Patient with any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.
- •Patients whose parents have not accepted the treatment regimen after explanation of it.
- •Contraindication to study drug mentioned in SmPC (Summary of Products Characteristics) of the study drugs.
- •Inclusion in another experimental anti-cancer drug therapy.
- •Study 1 non-inclusion criteria:
- •Any contraindication or concomitant disease that would preclude the Study 1 treatment procedure and could delay treatment.
- •These patients should be eligible for Study 2.
研究组 & 干预措施
Study 1: Melphalan or Melphalan + Topotecan
Randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
干预措施: Melphalan or Melphalan + Topotecan (Drug)
Study 1: Melphalan or Melphalan + Topotecan
Randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
干预措施: Thermotherapy (local treatment) (Device)
Study 1: Melphalan or Melphalan + Topotecan
Randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
干预措施: Cryotherapy (local treatment) (Device)
Study 1: Melphalan or Melphalan + Topotecan
Randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
干预措施: Iodine-125 plaques (local treatment) (Device)
Study 1: Melphalan or Melphalan + Topotecan
Randomized phase-II study evaluating the efficacy of Intra-Arterial Chemotherapy (IAC) with melphalan and topotecan versus melphalan alone, both in association with ophthalmologic treatments.
干预措施: Intravitreal Melphalan chemotherapy injections (local treatment) (Drug)
Study 2: Etoposide, carboplatin and vincristine
Neoadjuvant chemotherapy involves 2 to 6 cycles of combined etoposide, carboplatin and vincristine in association with ophthalmologic treatments.
干预措施: etoposide, carboplatin and vincristine or local ophthalmological treatment without IV chemotherapy (Drug)
Study 2: Etoposide, carboplatin and vincristine
Neoadjuvant chemotherapy involves 2 to 6 cycles of combined etoposide, carboplatin and vincristine in association with ophthalmologic treatments.
干预措施: Carboplatin administered on Day 1 (Drug)
Study 2: Etoposide, carboplatin and vincristine
Neoadjuvant chemotherapy involves 2 to 6 cycles of combined etoposide, carboplatin and vincristine in association with ophthalmologic treatments.
干预措施: Thermotherapy (local treatment) (Device)
Study 2: Etoposide, carboplatin and vincristine
Neoadjuvant chemotherapy involves 2 to 6 cycles of combined etoposide, carboplatin and vincristine in association with ophthalmologic treatments.
干预措施: Cryotherapy (local treatment) (Device)
Study 2: Etoposide, carboplatin and vincristine
Neoadjuvant chemotherapy involves 2 to 6 cycles of combined etoposide, carboplatin and vincristine in association with ophthalmologic treatments.
干预措施: Iodine-125 plaques (local treatment) (Device)
Study 2: Etoposide, carboplatin and vincristine
Neoadjuvant chemotherapy involves 2 to 6 cycles of combined etoposide, carboplatin and vincristine in association with ophthalmologic treatments.
干预措施: Intravitreal Melphalan chemotherapy injections (local treatment) (Drug)
结局指标
主要结局
Visual function
时间窗: when patient is 6 years old and at least 24 months of follow-up after study inclusion
Percentage of patients with major, mild or no impairment of visual function according to WHO criteria, i.e. normal bilateral visual acuity (≥ 6/10) or mild bilateral visual impairment (3/10 to 5/10) according to tumor location and extension
Study 1: Rate of eye preservation
时间窗: 24 months after randomization
Rate of eye preservation in the 2 treatments' arms
次要结局
- Ocular toxicity(Up to 24 months after inclusion in the study)
- Second malignant tumor(24 months after study inclusion)
- Additional assessment of visual function(In the year patients turn 6 years of age and at least 24 months of treatment)
- Systemic toxicity(Up to 24 months after inclusion in the study)
- Retinal assessment(In the year patients turn 6 years of age and at least 24 months of treatment)
- Relapse(within 24 months after inclusion)
- Integration at school(In the year patients turn 6 years of age and at least 24 months of treatment)