HCRN Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants
ESTHI
Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants
2 other identifiers
interventional
176
2 countries
21
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jul 2020
Longer than P75 for phase_3
21 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 27, 2019
CompletedFirst Posted
Study publicly available on registry
November 26, 2019
CompletedStudy Start
First participant enrolled
July 21, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 31, 2027
June 11, 2025
June 1, 2025
6.9 years
October 27, 2019
June 5, 2025
Conditions
Keywords
Outcome Measures
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.
12 months post randomized surgical intervention
Secondary Outcomes (2)
Bayley Scale of Infant Development-IV (Bayley-IV) Language Scaled Score
12 months post randomized surgical intervention
Bayley Scale of Infant Development-IV (Bayley-IV) Motor Scaled Score
12 months post randomized surgical intervention
Other Outcomes (19)
Vineland-3 Communication Domain Score
12 months post randomized surgical intervention
Vineland-3 Daily Living Skills Domain Score
12 months post randomized surgical intervention
Vineland-3 Socialization Domain Score
12 months post randomized surgical intervention
- +16 more other outcomes
Study Arms (2)
ETV+CPC
ACTIVE COMPARATORSubjects randomized to this arm will undergo an ETV+CPC procedure for treatment of Hydrocephalus
Ventriculoperitoneal Shunt
ACTIVE COMPARATORSubjects randomized to this arm will undergo a Ventriculoperitoneal Shunt procedure for treatment of Hydrocephalus
Interventions
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.
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.
Eligibility Criteria
You may qualify if:
- Corrected age \<104 weeks and 0 days,
- AND
- Child is ≥ 37 weeks post menstrual age,
- AND
- 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
- +1 more criteria
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- University of Utahlead
- University of Alabama at Birminghamcollaborator
- University of British Columbiacollaborator
- University of Pittsburghcollaborator
- The Hospital for Sick Childrencollaborator
- Seattle Children's Hospitalcollaborator
- Vanderbilt University Medical Centercollaborator
- Washington University School of Medicinecollaborator
- Nationwide Children's Hospitalcollaborator
- Johns Hopkins Universitycollaborator
- University of Calgarycollaborator
- University of Colorado, Denvercollaborator
- Children's Hospital Los Angelescollaborator
- National Institutes of Health (NIH)collaborator
- Hydrocephalus Associationcollaborator
- Penn State Universitycollaborator
- National Institute of Neurological Disorders and Stroke (NINDS)collaborator
- Baylor College of Medicinecollaborator
- University of Floridacollaborator
- Orlando Health, Inc.collaborator
- Virginia Commonwealth Universitycollaborator
- Trustees of Indiana Universitycollaborator
Study Sites (21)
Children's of Alabama
Birmingham, Alabama, 35233, United States
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
Children's Hospital of Los Angeles
Los Angeles, California, 90027, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Yale University
New Haven, Connecticut, 06520, United States
Wolfson Children's Hospital
Jacksonville, Florida, 32207, United States
Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
Trustees of Indiana University
Indianapolis, Indiana, 46202, United States
Johns Hopkins Children's Center
Baltimore, Maryland, 21287, United States
St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
The Pennsylvania State University
University Park, Pennsylvania, 16802, United States
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Primary Children's Hospital
Salt Lake City, Utah, 84118, United States
Virginia Commonwealth University
Richmond, Virginia, 23284, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
John Kestle, MD
University of Utah
- PRINCIPAL INVESTIGATOR
Abhaya Kulkarni, MD
University of Toronto
- PRINCIPAL INVESTIGATOR
David Limbrick, MD
Washington University School of Medicine
- PRINCIPAL INVESTIGATOR
Richard Holubkov, PhD
University of Utah
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- MD, Vice Chair of Clinical Research
Study Record Dates
First Submitted
October 27, 2019
First Posted
November 26, 2019
Study Start
July 21, 2020
Primary Completion (Estimated)
May 30, 2027
Study Completion (Estimated)
August 31, 2027
Last Updated
June 11, 2025
Record last verified: 2025-06
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP
- Time Frame
- Within one year of primary publication or within 18 months of the last study visit of the last subject, whichever occurs first.
After subject enrollment and 5 year follow up have been completed, we will prepare a final study database for analysis. A releasable database will be produced and completely de-identified in accordance with the definitions provided in the Health insurance Portability and Accountability Act (HIPAA). Namely, all identifiers specified in HIPAA will be recoded in a manner that will make it impossible to deduce or impute the specific identity of any patient. The database will not contain any institutional identifiers. We will also prepare a data dictionary that provides a concise definition of every data element included in the database. If specific data elements have idiosyncrasies that might affect interpretation or analysis, this will be discussed in the dictionary document. In accordance with policies determined by the investigators and funding sponsors, the releasable database will be provided to users in electronic form.