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Active, not recruitingNCT01425944Updated Mar 2, 2026

Innovative Approaches to Gauge Progression of Sturge-Weber Syndrome

An observational study in Sturge-Weber Syndrome, sponsored by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.. Active, not recruiting at 7 sites in United States. Open to participants aged 1 Month and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-03-02.

Sponsored by Hugo W. Moser Research Institute at Kennedy Krieger, Inc. · Observational

Study type
Observational
Model
Other
Time perspective
Cross-sectional
Enrollment
600
Ages
1 Month and older
Sex
All
01

Study summary

This study has three aims that hope to expand the knowledge on the cause of Sturge-Weber Syndrome (SWS) and improve clinical care of Sturge-Weber Syndrome patients.

Read the detailed description

This study is one of three projects of an NIH Rare Disease Clinical Research Consortium focused on brain blood vessel malformations in three different rare diseases. The focus of this project is on Sturge-Weber Syndrome.

We plan to improve the future understanding and treatment of Sturge-Weber Syndrome by 1) establishing a national consortium database which will gather lager amounts of clinical data and serve indirectly as a registry to foster future clinical trials and determine the usefulness of urine vascular biomarkers to determine the vascular remodeling of the SWS birthmark and choroidal angioma, 2) study vascular remodeling with retrospective and prospective neuroimaging to determine the vascular remodeling of the deep draining intraparenchymal vessels as it relates to SWS neurologic status, and 3) relate the GNAQ mutation to altered phosphorylation of pathway proteins and angiogenesis factors in SWS tissue.

02

Conditions studied

  • Sturge-Weber Syndrome

Keywords

  • Sturge Weber Syndrome
  • Biomarkers
  • DNA arrays
  • brain vessel malformations
03

Who can participate

Ages eligible
1 Month and older
Sexes eligible
All
Accepts healthy volunteers
Yes
Sampling method
Non-probability sample

Study population

For Aim 1, the population will be subjects with Sturge-Weber Syndrome and diagnosed brain involvement. There will be a separate group made up of family members of those with Sturge-Weber syndrome brain involvement to have as a control for the urine portion of Aim 1. For the optical coherence tomography (OCT) portion of Aim 1, the population will be subjects with Sturge-Weber Syndrome eye involvement. For Aim 2, the population will be subjects that have Sturge-Weber Syndrome with brain involvement. For Aim 3, the population will be subjects with Sturge-Weber Syndrome, diagnosed brain involvement, and V1 distribution Port-Wine Stain.

Inclusion criteria

For Aim 1:

For main sample:

  • Sturge-Weber syndrome
  • Diagnosed brain Involvement

For Control:

  • Family member of participating SWS patient

For OCT:

  • Sturge-Weber syndrome eye involvement

For Aim 2:

  • Sturge-Weber syndrome
  • Diagnosed Brain Involvement

For Aim 3:

  • Sturge-Weber syndrome
  • Diagnosed brain Involvement
  • Port-Wine Stain in V1 and/or V2 areas of face.

Exclusion criteria

Exclusion Criteria:

  • Not Diagnosed with Sturge-Weber syndrome with brain Involvement (or eye involvement for OCT)

For Aim 1:

  • Family member must not have certain medical conditions. A list will be provided before consent is given.

For Aim 3:

  • Not Diagnosed with Sturge-Weber syndrome with brain Involvement
  • No Port-Wine Stain
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Study design

Observational model
Other
Time perspective
Cross-sectional
Enrollment
600 participants (estimated)
Biospecimen retention
Samples with dna
05

What researchers measure

Primary outcomes

  1. Aim 1

    Descriptive statistics for the national database, correlation between neurologic score and urine angiogenesis factor, and correlation between PWS (port-wine stain) attributes, urine vascular factors, and neuroscore

    Time frame: All 5 years

  2. Aim 2

    Correlation between neuroscore and degree of collateral venous vessel opening

    Time frame: All 5 years

  3. Aim 3

    Correlation between GNAQ mutation status and hyperphosphorylation in downstream proteins

    Time frame: All 5 years

06

Study locations

7 sites
  • Kennedy Krieger Institute
    Baltimore, Maryland 21205, United States
  • Wayne State University/Children's Hospital of Michigan
    Detroit, Michigan 48201, United States
  • New York University
    New York, New York 10016, United States
  • Cincinnati Children's Hospital
    Cincinnati, Ohio 45229, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
  • Wills Eye Institute
    Philadelphia, Pennsylvania 19107, United States
  • Baylor College of Medicine/Texas Children's Hospital
    Houston, Texas 77030, United States
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Registry details

Key details

Study ID
NCT01425944
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Collaborators
National Institutes of Health (NIH), University of California, San Francisco, National Institute of Neurological Disorders and Stroke (NINDS), Duke University, Children's Hospital of Michigan, Baylor College of Medicine, Wills Eye, Nationwide Children's Hospital, New York University, Children's Hospital Medical Center, Cincinnati
Responsible party
Anne Comi, MD (Principal Investigator, Director Sturge-Weber Center, Kennedy Krieger Institute, Professor Johns Hopkins University School of Medicine, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.) — Principal investigator
First posted
Aug 30, 2011
Start date
Sep 2010
Primary completion
Dec 2025
Completion
Feb 9, 2027 (estimated)
Last update
Mar 2, 2026

Study contacts

Anne M Comi, M.D.
principal investigator · Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Feb 2026. You cannot join it, but the record below documents what was studied.

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