Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 176
- 试验地点
- 42
- 主要终点
- Bayley Scale of Infant Development-IV (Bayley-IV) Cognitive Scale score
研究概览
简要总结
Hydrocephalus is a potentially debilitating neurological condition that primarily affects babies under a year of age and has traditionally been treated by inserting a shunt between the brain and the abdomen. A newer endoscopic procedure offers hope of shunt- free treatment that may reduce complications over a child's life, but it is not clear if the endoscopic procedure results in similar intellectual outcome as shunt. Therefore, the investigators propose a randomized trial to compare intellectual outcome and brain structural integrity between these two treatments, to help families make the best treatment decision for their baby.
详细描述
The ESTHI Trial is a multi-center randomized controlled trial (RCT) comparing endoscopic third ventriculostomy with choroid plexus cauterization (ETV+CPC) and shunt in infants with hydrocephalus. The study will leverage the infrastructure of the Hydrocephalus Clinical Research Network (HCRN), a committed group of 14 leading North American pediatric neurosurgical centers with a long track-record of successful collaborative clinical research and RCTs in hydrocephalus. Optimal cognitive outcome is the primary concern of families and will, therefore, be the primary outcome. Assessment of dMRI, a validated, non-invasive method of measuring white matter microstructural integrity and structural connectivity in the developing brain, will provide further insight into the developmental consequences of these two treatments. The results of the RCT will help families determine the optimal treatment of hydrocephalus for their child.
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
- 1 Day 至 104 Weeks（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Corrected age <104 weeks and 0 days,
- •Child is ≥ 37 weeks post menstrual age,
- •Child must have symptomatic hydrocephalus, defined as:
- •Ventriculomegaly on MRI (frontal-occipital horn ratio (FOR) >0.45, which approximates "moderate ventriculomegaly"), and at least one of the following:
- •Head circumference >98th percentile for corrected age with either bulging fontanelle or splayed sutures
- •Upgaze paresis/palsy (sundowning)
- •Papilledema
- •Tense pseudomeningocele or tense fluid along a track
- •Vomiting or irritability, with no other attributable cause
- •Bradycardias or apneas, with no other attributable cause
- •Intracranial pressure (ICP) monitoring showing persistent elevation of pressure with or without plateau waves
- •No prior history of shunt insertion or endoscopic third ventriculostomy (ETV) procedure (previous temporization devices and/or external ventricular drains permissible)
排除标准
- •Hydrocephalus due to intraventricular hemorrhage in a child born before 37 weeks gestational age; OR
- •Anatomy not suitable for ETV+CPC or anteriorly placed ventriculoperitoneal shunt defined as:
- •Moderate to severe prepontine adhesions on steady state free precession (SSFP) or T2 weighted fast (turbo) spin echo (FSE/TSE) MRI, which includes the following sequences: FIESTA, FIESTA-C, TrueFISP, CISS, Balanced FFE (bFFE), CUBE, SPACE, VISTA, IsoFSE, and 3D MVOX
- •Closure of one or both foramina of Monro
- •Thick floor of third ventricle (≥ 3mm)
- •Narrow third ventricle (<5mm)
- •Presence of scalp, bone, or ventricular lesions that make placement of an anterior shunt impracticable; OR
- •Underlying condition with a high chance of mortality within 12 months; OR
- •Hydrocephalus with loculated CSF compartments; OR
- •Peritoneal cavity not suitable for distal shunt placement; OR
- •Active CSF infection; OR
- •Hydranencephaly; OR
- •Child requires an intraventricular procedure (e.g. endoscopic biopsy) in addition to the initial first-time permanent procedure for the treatment of hydrocephalus.
结局指标
主要结局
Bayley Scale of Infant Development-IV (Bayley-IV) Cognitive Scale score
时间窗: 12 months post randomized surgical intervention
The primary objective is to determine, in infants \<104 weeks corrected age, with hydrocephalus requiring treatment at tertiary care pediatric neurosurgery centers in North America, if treatment with ETV+CPC compared to shunt results in non-inferior cognitive outcome at 12 months from surgery, as measured by Bayley-IV Cognitive Scale score with a non-inferiority margin of 1.5. Scaled scores range from 1-19. Higher scores indicate better outcomes. Scores will also be obtained at 3 and 5 years of age.
次要结局
- Bayley Scale of Infant Development-IV (Bayley-IV) Motor Scaled Score(12 months post randomized surgical intervention)
- Bayley Scale of Infant Development-IV (Bayley-IV) Language Scaled Score(12 months post randomized surgical intervention)
研究者
John Kestle
MD, Vice Chair of Clinical Research
University of Utah