Endoscopic versus Shunt Treatment of Hydrocephalusin Infants

Comparing Endoscopic and Shunt Treatments for Hydrocephalus in Infants

J
Jason Chu

Primary Investigator

Enrolling By Invitation
1 day - 104 weeks
All
Phase N/A
2 participants needed
1 Location

Brief description of study

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.

THIS STUDY IS ENROLLING BY INVITATION ONLY

Detailed description of study

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. This study is being conducted to see if there is a difference between Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC) and Ventriculoperitoneal Shunt (VPS) on brain function.
Participants will be randomized. There is a 50/50 chance of getting ETV+CPC and a 50/50 chance of getting VPS.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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hydrocephalus
  • Age: 1 day - 104 weeks
  • Gender: All

Inclusion Criteria:

  1. Corrected age <104 weeks and 0 days,

    AND

  2. Child is ≥ 37 weeks post menstrual age,

    AND

  3. 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)
    • CSF leak
    • 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

    AND

  4. No prior history of shunt insertion or endoscopic third ventriculostomy (ETV) procedure (previous temporization devices and/or external ventricular drains permissible)

Exclusion Criteria:

  1. Hydrocephalus due to intraventricular hemorrhage in a child born before 37 weeks gestational age; OR
  2. 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
  3. Underlying condition with a high chance of mortality within 12 months; OR
  4. Hydrocephalus with loculated CSF compartments; OR
  5. Peritoneal cavity not suitable for distal shunt placement; OR
  6. Active CSF infection; OR
  7. Hydranencephaly; OR
  8. Child requires an intraventricular procedure (e.g. endoscopic biopsy) in addition to the initial first-time permanent procedure for the treatment of hydrocephalus.

This study investigates treatments for hydrocephalus, a neurological condition affecting infants, where fluid builds up in the brain. Traditionally, a shunt is used to drain this fluid to another part of the body. The purpose of this study is to compare this method with a newer technique called endoscopic third ventriculostomy with choroid plexus cauterization (ETV+CPC) to see which better supports brain function.

Participants will be randomly assigned to receive either the ETV+CPC or the shunt treatment. This study will use dMRI, a non-invasive imaging technique, to measure the effects of these treatments on brain structure. The results will help determine the impact of each treatment on the brain's development.

  • Who can participate: Infants with a corrected age under 104 weeks who have symptomatic hydrocephalus may participate. Key eligibility includes having ventriculomegaly and no prior shunt or endoscopic procedures. Infants must not have hydrocephalus due to intraventricular hemorrhage or other specific anatomical conditions.
  • Study details: Participants will be assigned either the endoscopic procedure or the shunt. The endoscopic procedure involves using a scope to create an opening in the brain to relieve pressure. A shunt involves placing a tube to drain fluid from the brain. Both treatments aim to manage hydrocephalus effectively.
Updated on 21 Aug 2025. Study ID: SNEU-UTAH-ESTHI, 26437
Please visit our main page to search for other studies you may be interested in. If you need help finding a study or have any questions, please contact us at inhealth@iu.edu

Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only