Clinical Study of Modified Carboplatin/vincristine Chemotherapy Regimen for Visual Function Protection in Children with Optic Pathway Gliomas
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 75
- 试验地点
- 1
- 主要终点
- VA improvement rate
研究概览
简要总结
Optic pathway glioma (OPG) can result in visual deterioration. Symptomatic patients often report deficits in visual acuity (VA), visual field, visual-evoked potentials (VEPs), strabismus, proptosis, disc swelling, and other visual/neurological problems. VA itself remains one of the most important outcome measures for OPG patients, with various studies showing strong ties of VA level to overall quality of life and well-being . Maintenance of favorable VA and vision outcomes is of paramount importance in the management of OPG.
In terms of management of OPG, surgery and radiotherapy are used on a more limited basis because of location of the tumors and risk of secondary tumors, respectively. Tumor stabilization often prioritized, and chemotherapy is considered ideal for tumor stabilization in OPG, but vision is not always retained and may worsen in some cases, partially due to low radiographic efficacy and long time interval to response of the current chemotherapy regimen.
In the prior study, the investigators modified the traditional carboplatin combined with vincristine regimen by increasing the dose of carboplatin and combining with an anti-angiogenic drug. Of the 15 OPG patients, objective response rate was 80% and the time to response was only 3.3 months. 8 (53%) patients experienced an improvement in visual acuity during therapy and 6 (40%) were stable, which was higher than the historical studies.
This study was launched to further verify the clinical efficacy of the modified regimen and its effect on visual acuity improvement.
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 Months 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 3months and ≤21years;
- •Patients with optic pathway gliomas diagnosed by histopathology or characteristic brain MRI and clinical features;
- •Measurable lesions, surgical resection degree < 95% or postoperative residual tumor ≥1.5cm^2;
- •KPS score ≥50 (age >12 years) or Lansky score ≥50 (age ≤12 years);
- •Clinical symptoms such as decreased visual acuity, visual field defect, optic disc edema, exophthalmia, increased intracranial pressure, diencephalic syndrome, etc;
- •No dysfunction of major organs.
排除标准
- •MRI examination is not available.
- •Failing to comply with the visual examination.
- •H3K27 mutations, even histopathological grade 1/
- •Receiving any other investigational agent.
- •History of allergic reactions attributed to compounds of similar chemical or biologic composition to the drugs used in this study.
- •Patients who have received organ transplants.
- •Patients infected with HIV or treponema pallidum.
- •Suffering from serious cardiovascular disease;T wave inversion or elevation or ST segment changes.
- •Patients who had coagulation disorder and were being treated with thrombolytic or anticoagulant drugs. Patients with significant clinical bleeding symptoms or clear bleeding tendency occurred within 3 months before enrollment, such as gastrointestinal bleeding, hemorrhagic gastric ulcer, gastrointestinal perforation, baseline fecal occult blood ++ or above, intratumoral or intracranial bleeding, or vasculitis, etc. Arteriovenous thrombosis events (such as cerebrovascular accident (including transient ischemic attack, cerebral hemorrhage and cerebral infarction), deep vein thrombosis and pulmonary embolism) occurred within 6 months before enrollment.
- •Pregnant or breastfeeding.
- •Other conditions considered inappropriate by the researcher for inclusion.
研究组 & 干预措施
optic pathway glioma
干预措施: Carboplatin (Drug)
optic pathway glioma
干预措施: Vincristine (Drug)
optic pathway glioma
干预措施: Recombinant human endostatin (Drug)
结局指标
主要结局
VA improvement rate
时间窗: up to 3 years
Percentage of visual acuity improvement
次要结局
- time to VA improvement(up to 3 years)
- objective response rate(up to 3 years)
- median time to response(up to 3 years)
研究者
Junping Zhang
Chief physician
Beijing Sanbo Brain Hospital