HydrocephalusNCT03650101
Improving Infant Hydrocephalus Outcomes in Uganda
An observational study of Hydrocephalus, run by Boston Children's Hospital at 3 sites in 3 countries.
Trial summary
Neonatal postinfectious hydrocephalus (PIH) is a major public health problem in East Africa.The standard treatment has long been placement of a ventriculoperitoneal shunt (VPS) but these devices require life-long maintenance and nearly all fail multiple times. Read more about this study
- Who is it for?
- Ages 1 day to 180 days
- Conditions the study is for
- Hydrocephalus
- What is being tested?
- ETV/CPC
- Type of treatment
- Surgery or procedure
- How many people
- 400 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 5 May 2021to Dec 2027
- Phase
- Observational
- 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 180 days
- All sexes
- No healthy volunteers
- Child
Study population: Infants less than six months old with progressive hydrocephalus
Who may be able to join
- Infants less than 180 days (six months) old
- Symptomatic hydrocephalus characterized by abnormal rate of head growth, full anterior fontanel, ventriculomegaly
- A parent or a guardian qualified by Ugandan law to give informed consent
- Patients from Eastern, Central and Northern districts of Uganda, and in geographic proximity to CURE hospital will be eligible
Who cannot join
- Age greater than six months
- No evidence of progressive hydrocephalus
- Patients outside of the districts specified in the inclusion criteria
Trial locations and contacts
3 sites in 3 countries.
3 sites in 3 countries
United States1 site
Penn State University
University Park, Pennsylvania, 16802
About this study
Official title: Improving Infant Hydrocephalus Outcomes in Uganda: Predicting Developmental Outcomes and Identifying Patients at Risk for Early Treatment Failure After ETV/CPC
Neonatal postinfectious hydrocephalus (PIH) is a major public health problem in East Africa.The standard treatment has long been placement of a ventriculoperitoneal shunt (VPS) but these devices require life-long maintenance and nearly all fail multiple times. Endoscopic Third Ventriculostomy (ETV) with Choroid Plexus Cauterization (ETV/CPC) is an alternate treatment to give patients a shunt-free life. In this study, the investigators aim to optimize the metrics of evaluation as quantitative prognostic indicators of treatment response and long term outcomes.
- Conditions
- Hydrocephalus
- Being tested
- ETV/CPC
- Keywords
- Hydrocephalus
- Medical subject headings
- Hydrocephalus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ETV/CPCProcedure or surgery
The Endoscopic Third Ventriculostomy/Choroid Plexus Cauterization (ETV/CPC) will comprise a standard frontal approach with flexible endoscopy.
| Study group | Role | What they receive |
|---|---|---|
| Hydrocephalus group | Inclusion criteria:
1. Age less than six months
2. Symptomatic hydrocephalus characterized by abnormal rate of head growth, full anterior fontanel, ventriculomegaly
3. A parent or a guardian qualified by Ugandan law to give informed consent
4. Patients from Eastern, Central and Northern districts of Uganda, and in geographic proximity to CURE hospital will be eligible
Exclusion criteria:
1. Age greater than six months
2. No evidence of progressive hydrocephalus
3. Patients outside of the districts specified in the inclusion criteria Receives: Procedure: ETV/CPC | |
| Control group | Inclusion criteria: 1. Born at GA ≥ 37 weeks 2. Age less than six months at the time of enrollment 3. No known medical conditions 4. With a parent or a guardian qualified by Ugandan law to give informed consent 5. Parents live in one of the villages in the Mbale or Budaka District, in geographic proximity to CCHU. Exclusion criteria: 1. Born at GA < 37 weeks 2. Age greater than six months 3. Evidence of one or more medical conditions 4. Living outside of the districts specified in the inclusion criteria. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bayley Scales of Infant Development, Third Edition (BSID-3), cognitive scaled score
Scores on the BSID-3, which is used to evaluate infants and toddlers 1 to 42 months of age, range from 1 to 19, with higher scores indicating better performance; the mean (±standard deviation(SD)) score in the general population is 10±3.
Measured: 24 months of age
Incidence of ETV/CPC treatment failure
Treatment failure or success will be determined with the use of clinical and radiographic criteria.
- Treatment success will be determined as the shift in the growth of head circumference to a normal rate, as plotted on a standard growth chart; decompression of the anterior fontanel; relief of symptoms of elevated intracranial pressure, such as irritability and vomiting; resolution of down-gaze or sixth cranial nerve palsy; and a decrease or arrest in ventriculomegaly as determined on Computerized Tomography (CT).
- Treatment failure will be defined as treatment-related death or the need for a second operation for infection or for the recurrence of hydrocephalus.
Measured: 6 months post-treatment
Secondary outcomes (3)
Cerebral oxygen metabolism
cerebral oxygen metabolism will be measured with near-infrared spectroscopy
Measured: pre- and post-, 6, 12 months post-treatment and 24 months of age
brain volume
brain volume will be quantitatively estimated from the head CT scan
Measured: pre-, 6, 12 months post-treatment and 24 months of age
Bayley Scales of Infant Development, Third Edition (BSID-3), cognitive scaled score at 12 months post-treatment
Scores on the BSID-3, which is used to evaluate infants and toddlers 1 to 42 months of age, range from 1 to 19, with higher scores indicating better performance; the mean (±standard deviation) score in the general population is 10±3.
Measured: 12 months post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2018
- Study start5 May 2021
- Primary completion15 May 2025
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HydrocephalusStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 400 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- The Hospital for Sick Children, CURE Children's Hospital, Uganda, Yale University
- Responsible party
- Principal investigator, Pei-Yi Lin, Boston Children's Hospital
- Investigators
- Pei-Yi Lin, PhD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03650101
- Sponsor's protocol ID
- P00029806
- Status last verified
- Mar 2026
- First submitted
- 23 Aug 2018
- Last updated
- 18 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data will not be shared with researchers outside of the study team.
Publications
Vadset TA, Rajaram A, Hsiao CH, Kemigisha Katungi M, Magombe J, Seruwu M, Kaaya Nsubuga B, Vyas R, Tatz J, Playter K, Nalule E, Natukwatsa D, Wabukoma M, Neri Perez LE, Mulondo R, Queally JT, Fenster A, Kulkarni AV, Schiff SJ, Grant PE, Mbabazi Kabachelor E, Warf BC, Sutin JDB, Lin PY. Improving Infant Hydrocephalus Outcomes in Uganda: A Longitudinal Prospective Study Protocol for Predicting Developmental Outcomes and Identifying Patients at Risk for Early Treatment Failure after ETV/CPC. Metabolites. 2022 Jan 14;12(1):78. doi: 10.3390/metabo12010078.
PubMed 35050201
Study at a glance
From the registry record, last updated by the sponsor on 18 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Improving Infant Hydrocephalus Outcomes in Uganda: Predicting Developmental Outcomes and Identifying Patients at Risk for Early Treatment Failure After ETV/CPC
- ClinicalTrials.gov ID
- NCT03650101
- Acronym / sponsor ID
- P00029806
- Conditions
- Hydrocephalus
- Keywords
- Hydrocephalus
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 400 participants (estimated)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- The Hospital for Sick Children, CURE Children's Hospital, Uganda, Yale University
- Study start
- 5 May 2021 (actual)
- Primary completion
- 15 May 2025 (actual)
- Study completion
- Dec 2027 (estimated)
- First posted
- 28 Aug 2018 (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.
Neonatal postinfectious hydrocephalus (PIH) is a major public health problem in East Africa.The standard treatment has long been placement of a ventriculoperitoneal shunt (VPS) but these devices require life-long maintenance and nearly all fail multiple times. Endoscopic Third Ventriculostomy (ETV) with Choroid Plexus Cauterization (ETV/CPC) is an alternate treatment to give patients a shunt-free life. In this study, the investigators aim to optimize the metrics of evaluation as quantitative prognostic indicators of treatment response and long term outcomes.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hydrocephalus group | – | Procedure: ETV/CPCInclusion criteria: 1. Age less than six months 2. Symptomatic hydrocephalus characterized by abnormal rate of head growth, full anterior fontanel, ventriculomegaly 3. A parent or a guardian qualified by Ugandan law to give informed consent 4. Patients from Eastern, Central and Northern districts of Uganda, and in geographic proximity to CURE hospital will be eligible Exclusion criteria: 1. Age greater than six months 2. No evidence of progressive hydrocephalus 3. Patients outside of the districts specified in the inclusion criteria |
| Control group | – | –Inclusion criteria: 1. Born at GA ≥ 37 weeks 2. Age less than six months at the time of enrollment 3. No known medical conditions 4. With a parent or a guardian qualified by Ugandan law to give informed consent 5. Parents live in one of the villages in the Mbale or Budaka District, in geographic proximity to CCHU. Exclusion criteria: 1. Born at GA < 37 weeks 2. Age greater than six months 3. Evidence of one or more medical conditions 4. Living outside of the districts specified in the inclusion criteria. |
Who may qualify
- Ages
- 1 day to 180 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Infants less than six months old with progressive hydrocephalus
Key inclusion criteria (4 listed)
- Infants less than 180 days (six months) old
- Symptomatic hydrocephalus characterized by abnormal rate of head growth, full anterior fontanel, ventriculomegaly
- A parent or a guardian qualified by Ugandan law to give informed consent
- Patients from Eastern, Central and Northern districts of Uganda, and in geographic proximity to CURE hospital will be eligible
Key exclusion criteria (3 listed)
- Age greater than six months
- No evidence of progressive hydrocephalus
- Patients outside of the districts specified in the inclusion criteria
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 |
|---|---|---|
| ETV/CPCThe Endoscopic Third Ventriculostomy/Choroid Plexus Cauterization (ETV/CPC) will comprise a standard frontal approach with flexible endoscopy. | Procedure or surgery | Hydrocephalus group |
Primary outcome
Bayley Scales of Infant Development, Third Edition (BSID-3), cognitive scaled score
Time frame: 24 months of age
Scores on the BSID-3, which is used to evaluate infants and toddlers 1 to 42 months of age, range from 1 to 19, with higher scores indicating better performance; the mean (±standard deviation(SD)) score in the general population is 10±3.
Incidence of ETV/CPC treatment failure
Time frame: 6 months post-treatment
Treatment failure or success will be determined with the use of clinical and radiographic criteria. * Treatment success will be determined as the shift in the growth of head circumference to a normal rate, as plotted on a standard growth chart; decompression of the anterior fontanel; relief of symptoms of elevated intracranial pressure, such as irritability and vomiting; resolution of down-gaze or sixth cranial nerve palsy; and a decrease or arrest in ventriculomegaly as determined on Computerized Tomography (CT). * Treatment failure will be defined as treatment-related death or the need for a second operation for infection or for the recurrence of hydrocephalus.
3 secondary outcomes
Cerebral oxygen metabolism
Time frame: pre- and post-, 6, 12 months post-treatment and 24 months of age
brain volume
Time frame: pre-, 6, 12 months post-treatment and 24 months of age
Bayley Scales of Infant Development, Third Edition (BSID-3), cognitive scaled score at 12 months post-treatment
Time frame: 12 months post-treatment
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 18 Mar 2026.
- Results publication: PubMed 35050201Vadset TA, Rajaram A, Hsiao CH, Kemigisha Katungi M, Magombe J, Seruwu M, Kaaya Nsubuga B, Vyas R, Tatz J, Playter K, Nalule E, Natukwatsa D, Wabukoma M, Neri Perez LE, Mulondo R, Queally JT, Fenster A, Kulkarni AV, Schiff SJ, Grant PE, Mbabazi Kabachelor E, Warf BC, Sutin JDB, Lin PY. Improving Infant Hydrocephalus Outcomes in Uganda: A Longitudinal Prospective Study Protocol for Predicting Developmental Outcomes and Identifying Patients at Risk for Early Treatment Failure after ETV/CPC. Metabolites. 2022 Jan 14;12(1):78. doi: 10.3390/metabo12010078.
- Individual participant dataSharing planned: No. Individual participant data will not be shared with researchers outside of the study team.
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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.
Pei-Yi Lin, PhD principal investigator
Boston Children's Hospital
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hydrocephalus, run by Boston Children's Hospital at 3 sites in 3 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 4 inclusion and 3 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 18 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Canada, Uganda. 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 Scales of Infant Development, Third Edition (BSID-3), cognitive scaled score (time frame: 24 months of age). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03650101, which also lists 1 linked publication.
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 18 Mar 2026.