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Sturge - Weber Syndrome (SWS)Phase 2Epi-PreNCT07778810

Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber Syndrome

A Phase 2 study testing Epidiolex 100 mg/mL Oral Solution in people with Sturge - Weber Syndrome (SWS), run by Johns Hopkins University at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

Patients with Sturge-Weber syndrome (SWS) are frequently affected by seizures, and seizures are associated with poorer neurological outcomes. To date there is no established means of predicting or preventing seizure onset. Cannabidiol (Epidiolex) was well tolerated in an open label study in this population. Read more about this study

Who is it for?
Ages 1 month to 18 months
Conditions the study is for
Sturge - Weber Syndrome (SWS)
What is being tested?
Epidiolex 100 mg/mL Oral Solution
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited States
Study dates
17 Sep 2026to Jul 2028
Phase
Phase 2
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 month to 18 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Clinical diagnosis of Sturge-Weber Syndrome.
  • Age 1 to 18 months of age, inclusive.
  • Neuroimaging demonstrating involvement of 3 or more lobes, or bilateral involvement.
  • No history of seizures.
  • Patient's parent or legal guardian provides written informed consent prior to treatment initiation.

Who cannot join

  • Any severe and/or uncontrolled medical conditions at randomization including, but not limited to the following:
  • Liver disease such as cirrhosis, decompensated liver disease, and chronic hepatitis (i.e. quantifiable HBVDNA and/or positive HbsAg, quantifiable HCV-RNA). Specifically the patient should not have either AST or ALT more than 1.5 times the upper limit of normal. The Child- Pugh Score must be A (mild) which is a score of 5-6 (minimum/normal score=5). Child-Turcotte-Pugh (CTP) Calculator - Clinical Calculators - Hepatitis C Online (uw.edu)
  • Uncontrolled diabetes as defined by fasting serum glucose > 1.5 ULN
  • Active (acute or chronic) or uncontrolled severe infections
  • Active, bleeding diathesis
  • Any other neurological diagnosis that increases the risk of seizure or neurodevelopmental disability.
Show all 13Show fewer
  • Patients who have had a major surgery or significant traumatic injury within 4 weeks of study entry, patients who have not recovered from the side effects of any major surgery (defined as requiring general anesthesia), or patients that may require major surgery during the course of the study.
  • Prior treatment with any investigational drug or use of any other cannabis product within the preceding 4 weeks prior to study entry.
  • Use of aspirin.
  • Allergic reaction/hypersensitivity to Epidiolex or other forms of cannabidiol.
  • Concern for non-compliance to medical regimens, or concern that the patient will not be able to complete the entire study, whether due to reliability or logistical barriers. This includes those in foster care, or those unable to keep follow-up appointments, maintain close contact with Principal Investigator, or complete all necessary studies to maintain safety.
  • Use of any seizure medication (whether for seizures or any other reason) (e.g. valproate, clobazam).
  • Use of chronic medications other than a multi-vitamin and/or vitamin D supplements.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Anne Comi, MD contact

443-923-9172

comi@kennedykrieger.org

Brenna Keam, BS contact

443-923-9127

keam@kennedykrieger.org

United States1 site, 1 recruiting

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205

    Anne Comi, MD, 443-923-9172, comi@kennedykrieger.org

    Brenna Keam, BS, 443-923-9127, keam@kennedykrieger.org

    Recruiting

Other trials in United States

About this study

Official title: Epidiolex Pilot Trial for Presymptomatic Treatment of Sturge-Weber Syndrome

Patients with Sturge-Weber syndrome (SWS) are frequently affected by seizures, and seizures are associated with poorer neurological outcomes. To date there is no established means of predicting or preventing seizure onset. Cannabidiol (Epidiolex) was well tolerated in an open label study in this population. This trial will evaluate whether Epidiolex in presymptomatic Sturge-Weber patients may delay the onset of seizures and improve neurological outcome.

Read the detailed description

This is an open-label, prospective dose escalation study to determine the safety and tolerability of presymptomatic treatment of SWS patients with Epidiolex. The investigators hypothesize that Epidiolex will be well tolerated and delay seizure onset in SWS patients. The total number of subjects to be consented for this safety study=10 babies with SWS; the initial funded phase will be with 6 subjects. Interim analysis will be completed after 6 subjects; the remaining 4 will be enrolled as warranted for 6 months on Epidiolex. Subjects will be dosed as recommended clinically (starting at 2.5 mg/kg/day divided bid, with weekly increases over the next 4 weeks to a maximum dose of 20 mg/kg/day) as tolerated. Neurologic visits during the trial, the bloodwork, EEGs, and motor assessments will be done as research procedures. Once the first dose of the study drug is administered, a blood draw will be done at 4-hour and 12-hour post study drug administration. A mini-PK will be similarly performed once the maximum maintenance dose is achieved.

Being tested
Epidiolex 100 mg/mL Oral Solution
Keywords
Presymptomatic, Open-label, Cannabidiol
Medical subject headings
Brain Stem Infarctions

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Epidiolex 100 mg/mL Oral SolutionDrug

    Also called Cannabidiol, CBD

    Epidiolex is a prescription form of cannabidiol used to treat seizures. Epidiolex will be started at 2.5 mg/kg/day divided twice daily. The dose will increase weekly as tolerated over the next 5 weeks to a target dose of 20 mg/kg/day.

Study groups in NCT07778810
Study groupRoleWhat they receive
Presymptomatic Epidiolex TreatmentExperimentalEpidiolex will be started at 2.5 mg/kg/day divided twice daily. The dose will increase weekly as tolerated over the next 5 weeks to a target dose of 20 mg/kg/day.
Receives: Drug: Epidiolex 100 mg/mL Oral Solution

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safe completion as assessed by the number of participants with no significant side effects

    Measured: 6 months

  2. Safe completion as assessed by the number of participants with no seizure onset

    Measured: 6 months

Secondary outcomes (5)

  1. Sturge-Weber Syndrome Neuroscore (SWS-NRS)

    Neuroscore is a published SWS ordinal score that incorporates domains of visual field cut (0-2), hemiparesis (0-4), seizure frequency (0-4), and cognitive function (0-5). Total score is sum of 4 ratings with a total score from 0-15. Higher score worse impairment. This will be obtained at each study visit.

    Measured: baseline, week 5, week 12, and week 24

  2. Milestone checklist

    A publicly available milestone checklist from Pathways.org will be completed at every study visit. The checklist is personalized based on age. Does not use a numerical or point based scoring system. It uses a pass/fail or completed/not-completed checklist tracking format related to Motor, Sensory, Communication, and Feeding abilities.

    Measured: baseline, week 5, week 12, and week 24

  3. Modified Early Motor Questionnaire (EMQ)

    The Modified EMQ will be performed at baseline and at week 24. The EMQ evaluates abilities in three domains: Gross Motor skills, Fine Motor Skills, and Perception-Action Integration. It uses a 5-point scale including -2 = Sure that child does NOT show behavior, -1 = Child probably does NOT show behavior yet, 0 = Unsure whether child could do this or not, 1 = Child probably shows this behavior, and 2 = Sure that child shows this behavior and remember a particular instance. Minimum total score is -200. Maximum total score is 200. Higher score means higher certainty in the child's behaviors.

    Measured: Baseline, week 24

  4. Modified Erhardt Developmental Prehension Assessment (EDPA)

    The Modified EDPA will be performed at baseline and at week 24. The EDPA will evaluate hand skills and arm-hand movements at baseline and in response to the study drug. Both right and left hands are tested. Individual test components are scored by a well-established pattern (+), pattern not yet achieved (-), and temporary pattern to be replaced by a more mature pattern (++). Maximum score would be a well-established pattern (+) for all EDPA items. Higher score better, means a greater number of well-established hand skills and arm-hand movements.

    Measured: Baseline, week 24

  5. Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT)

    The PEDI-CAT will be performed at baseline and at week 24. The PEDI-CAT will evaluate abilities in four functional domains: Daily Activities, Mobility, Social/Cognitive, and Responsibility. The PEDI-CAT uses a 4-point scale including 1 = Unable, 2 = Hard, 3 = A Little Hard, and 4 = Easy. Minimum scaled score is 20. Maximum scaled score is 80. Higher total score means better functional ability.

    Measured: Baseline, week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Aug 2026
  2. Study start17 Sep 2026
  3. Primary completionJul 2028 (estimated)
  4. Study completionJul 2028 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Open-label
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07778810
Sponsor's protocol ID
IRB00326943
Acronym
Epi-Pre
Status last verified
Oct 2026
First submitted
18 Aug 2026
Last updated
2 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Demographics, baseline assessments, completion assessments, adverse events.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Epidiolex 100 mg/mL Oral Solution in people with Sturge - Weber Syndrome (SWS), run by Johns Hopkins University at 1 site in United States.

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 5 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: looking for participants now, as of the sponsor's last update on 2 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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?

Contact Anne Comi, MD (443-923-9172, comi@kennedykrieger.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safe completion as assessed by the number of participants with no significant side effects (time frame: 6 months). 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 NCT07778810.

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 2 Oct 2026.

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