HydrocephalusESTHINCT04177914
HCRN Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants
A study testing Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC) and Ventriculoperitoneal Shunt in people with Hydrocephalus, run by University of Utah at 21 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 1 day to 104 weeks
- Conditions the study is for
- Hydrocephalus
- What is being tested?
- Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC) and Ventriculoperitoneal Shunt
- Type of treatment
- Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 165 participants
- Where
- 21 sitesin 2 countries
- Study dates
- 21 Jul 2020to 31 Aug 2027
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 104 weeks
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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:
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- 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
- No prior history of shunt insertion or endoscopic third ventriculostomy (ETV) procedure (previous temporization devices and/or external ventricular drains permissible)
Who cannot join
- 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)
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- 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.
Trial locations and contacts
21 sites in 2 countries.
21 sites in 2 countries
United States18 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins Children's Center
Baltimore, Maryland, 21287
Children's of Alabama
Birmingham, Alabama, 35233
Nationwide Children's Hospital
Columbus, Ohio, 43205
Texas Children's Hospital
Houston, Texas, 77030
Trustees of Indiana University
Indianapolis, Indiana, 46202
Wolfson Children's Hospital
Jacksonville, Florida, 32207
Children's Hospital of Los Angeles
Los Angeles, California, 90027
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232
Yale University
New Haven, Connecticut, 06520
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Phoenix Children's Hospital
Phoenix, Arizona, 85016
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Virginia Commonwealth University
Richmond, Virginia, 23284
Primary Children's Hospital
Salt Lake City, Utah, 84118
Seattle Children's Hospital
Seattle, Washington, 98105
St. Louis Children's Hospital
St Louis, Missouri, 63110
The Pennsylvania State University
University Park, Pennsylvania, 16802
Canada3 sites
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
About this study
Official title: Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants
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.
Read the detailed description
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.
- Conditions
- Hydrocephalus
- Being tested
- Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC), Ventriculoperitoneal Shunt
- Keywords
- Hydrocephalus, Infants, Ventriculoperitoneal Shunt, ETV+CPC, endoscopic third ventriculostomy, choroid plexus cauterization
- Medical subject headings
- Hydrocephalus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC)Procedure or 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 ShuntDevice
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.
| Study group | Role | What they receive |
|---|---|---|
| ETV+CPC | Active comparator | Subjects randomized to this arm will undergo an ETV+CPC procedure for treatment of Hydrocephalus Receives: Procedure: Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC) |
| Ventriculoperitoneal Shunt | Active comparator | Subjects randomized to this arm will undergo a Ventriculoperitoneal Shunt procedure for treatment of Hydrocephalus Receives: Device: Ventriculoperitoneal Shunt |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 12 months post randomized surgical intervention
Secondary outcomes (2)
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.
Measured: 12 months post randomized surgical intervention
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.
Measured: 12 months post randomized surgical intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Nov 2019
- Study start21 Jul 2020
- Primary completion30 May 2027 (estimated)
- Study completion31 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HydrocephalusStudies of the same condition, open and under way.
- More trials run by University of UtahThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 165 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Utah
- Sponsor type
- University, hospital or other
- Collaborators
- University of Alabama at Birmingham, University of British Columbia, University of Pittsburgh, The Hospital for Sick Children, Seattle Children's Hospital, Vanderbilt University Medical Center, Washington University School of Medicine, Nationwide Children's Hospital, Johns Hopkins University, University of Calgary, University of Colorado, Denver, Children's Hospital Los Angeles, National Institutes of Health (NIH), Hydrocephalus Association, Penn State University, National Institute of Neurological Disorders and Stroke (NINDS), Baylor College of Medicine, Orlando Health, Inc., Virginia Commonwealth University, Trustees of Indiana University
- Responsible party
- Principal investigator, John Kestle, University of Utah
- Investigators
- John Kestle, MD, Study chair, University of UtahAbhaya Kulkarni, MD, Principal investigator, University of TorontoDavid Limbrick, MD, Principal investigator, Washington University School of MedicineRichard Holubkov, PhD, Principal investigator, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04177914
- Sponsor's protocol ID
- HCRN 012
- Other IDs
- 1U01NS107486-01A1
- Acronym
- ESTHI
- Status last verified
- Sep 2026
- First submitted
- 27 Oct 2019
- Last updated
- 25 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Endoscopic Versus Shunt Treatment of Hydrocephalus in Infants
- ClinicalTrials.gov ID
- NCT04177914
- Acronym / sponsor ID
- ESTHI / HCRN 012
- Conditions
- Hydrocephalus
- Keywords
- Hydrocephalus, Infants, Ventriculoperitoneal Shunt, ETV+CPC, endoscopic third ventriculostomy, choroid plexus cauterization
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 165 participants (actual)
- Lead sponsor
- University of UtahUniversity, hospital or other
- Collaborators
- University of Alabama at Birmingham, University of British Columbia, University of Pittsburgh, The Hospital for Sick Children, Seattle Children's Hospital, Vanderbilt University Medical Center, Washington University School of Medicine, Nationwide Children's Hospital, Johns Hopkins University, University of Calgary, University of Colorado, Denver, Children's Hospital Los Angeles, National Institutes of Health (NIH), Hydrocephalus Association, Penn State University, National Institute of Neurological Disorders and Stroke (NINDS), Baylor College of Medicine, Orlando Health, Inc., Virginia Commonwealth University, Trustees of Indiana University
- Study start
- 21 Jul 2020 (actual)
- Primary completion
- 30 May 2027 (estimated)
- Study completion
- 31 Aug 2027 (estimated)
- First posted
- 26 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ETV+CPC | Active comparator | Procedure: Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC)Subjects randomized to this arm will undergo an ETV+CPC procedure for treatment of Hydrocephalus |
| Ventriculoperitoneal Shunt | Active comparator | Device: Ventriculoperitoneal ShuntSubjects randomized to this arm will undergo a Ventriculoperitoneal Shunt procedure for treatment of Hydrocephalus |
Who may qualify
- Ages
- 1 day to 104 weeks
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (16 listed)
- Corrected age <104 weeks and 0 days,
- AND
- Child is ≥ 37 weeks post menstrual age,
- AND
- Child must have symptomatic hydrocephalus, defined as:
Key exclusion criteria (13 listed)
- 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:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC)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. | Procedure or surgery | ETV+CPC |
| Ventriculoperitoneal ShuntThe 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. | Device | Ventriculoperitoneal Shunt |
Primary outcome
Bayley Scale of Infant Development-IV (Bayley-IV) Cognitive Scale score
Time frame: 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.
2 secondary outcomes
Bayley Scale of Infant Development-IV (Bayley-IV) Language Scaled Score
Time frame: 12 months post randomized surgical intervention
Bayley Scale of Infant Development-IV (Bayley-IV) Motor Scaled Score
Time frame: 12 months post randomized surgical intervention
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2026.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John Kestle, MD study chair
University of Utah
Abhaya Kulkarni, MD principal investigator
University of Toronto
David Limbrick, MD principal investigator
Washington University School of Medicine
21 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Endoscopic Third Ventriculostomy with Choroid Plexus Cauterization (ETV+CPC) and Ventriculoperitoneal Shunt in people with Hydrocephalus, run by University of Utah at 21 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 25 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bayley Scale of Infant Development-IV (Bayley-IV) Cognitive Scale score (time frame: 12 months post randomized surgical intervention). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2026.