HCRN Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants

ConditionHydrocephalus
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age1-104
SponsorUniversity of Utah

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Corrected age <104 weeks and 0 days,

Child is ≥ 37 weeks post menstrual age,

Head circumference >98th percentile for corrected age with either bulging fontanelle or splayed sutures

Upgaze paresis/palsy (sundowning)

Disqualifiers

Hydrocephalus due to intraventricular hemorrhage in a child born before 37 weeks gestational age; OR

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)

Trial design

Design model

Parallel

Treatments tested in this trial

  • Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC)

    Procedure/Surgery

    Since the early 1990s, ETV has become the main alternative to shunting for hydrocephalus. This procedure involves placing an endoscopic camera into the ventricles of the brain and creating a hole in the floor of the third ventricle to act as an internal bypass for obstructed CSF. The cauterization of choroid plexus (CPC) involves the use of a device to burn or cauterize tissue from the choroid plexus. The choroid plexus of the brain exists in the lateral ventricles, the third ventricle, and the fourth ventricle. Its main role is the production of CSF. The success of ETV alone is poor in infants, but when combined with CPC, improved results have been observed and ETV+CPC has become a safe viable option for these children.

  • Ventriculoperitoneal Shunt

    Device

    The most common treatment for hydrocephalus has been the insertion of a ventriculoperitoneal shunt, which has been in popular use for over 50 years. This consists of silastic tubing attached to a valve mechanism that runs subcutaneously from the head to the abdomen. It is one of the most common procedures performed by pediatric neurosurgeons.

Treatment groups

176 Participants
are divided into 2 treatment groups
Group A: ETV+CPCActive comparator 1 intervention
Group B: Ventriculoperitoneal ShuntActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

Bayley Scale of Infant Development-IV (Bayley-IV) Cognitive Scale score

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.

Time frame
12 months post randomized surgical intervention

Secondary outcomes

1

Bayley Scale of Infant Development-IV (Bayley-IV) Language Scaled Score

To determine, in the same cohort of infants, if ETV+CPC compared to shunt results in non-inferior Bayley-IV Language Scaled scores. Scaled scores range from 1-19. Higher scores indicate better outcomes.

Time frame
12 months post randomized surgical intervention
2

Bayley Scale of Infant Development-IV (Bayley-IV) Motor Scaled Score

To determine, in the same cohort of infants, if ETV+CPC compared to shunt results in non-inferior Bayley-IV Motor Scaled scores. Scaled scores range from 1-19. Higher scores indicate better outcomes.

Time frame
12 months post randomized surgical intervention

Other outcomes

1

Vineland-3 Communication Domain Score

To determine, in the same cohort of infants, if ETV+CPC compared to shunt results in non-inferior Vineland-3 Communication Domain scores. Domain scores range from 20-140. Higher scores indicate better outcomes.

Time frame
12 months post randomized surgical intervention
2

Vineland-3 Daily Living Skills Domain Score

To determine, in the same cohort of infants, if ETV+CPC compared to shunt results in non-inferior Vineland-3 Daily Living Skills Domain scores. Domain scores range from 20-140. Higher scores indicate better outcomes.

Time frame
12 months post randomized surgical intervention
3

Vineland-3 Socialization Domain Score

To determine, in the same cohort of infants, if ETV+CPC compared to shunt results in non-inferior Vineland-3 Socialization Domain scores. Domain scores range from 20-140. Higher scores indicate better outcomes.

Time frame
12 months post randomized surgical intervention
4

Vineland-3 Motor Skills Domain Score

To determine, in the same cohort of infants, if ETV+CPC compared to shunt results in non-inferior Vineland-3 Motor Skills Domain scores. Domain scores range from 20-140. Higher scores indicate better outcomes.

Time frame
12 months post randomized surgical intervention

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Utah

Lead sponsor

University of Alabama at Birmingham

Collaborator

University of British Columbia

Collaborator

University of Pittsburgh

Collaborator

The Hospital for Sick Children

Collaborator

Seattle Children's Hospital

Collaborator

Vanderbilt University Medical Center

Collaborator

Washington University School of Medicine

Collaborator

Nationwide Children's Hospital

Collaborator

Johns Hopkins University

Collaborator

University of Calgary

Collaborator

University of Colorado, Denver

Collaborator

Children's Hospital Los Angeles

Collaborator

National Institutes of Health (NIH)

Collaborator

Hydrocephalus Association

Collaborator

Penn State University

Collaborator

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Baylor College of Medicine

Collaborator

University of Florida

Collaborator

Orlando Health, Inc.

Collaborator

Virginia Commonwealth University

Collaborator

Trustees of Indiana University

Collaborator