Presymptomatic Epidiolex for Sturge-Weber Syndrome
Official title: Epidiolex Pilot Trial for Presymptomatic Treatment of Sturge-Weber Syndrome
Run by Johns Hopkins University · NCT07778810 · Data updated 2026-11-12
This study tests whether cannabidiol (Epidiolex) started in babies with Sturge-Weber syndrome can delay the first seizure.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | 1 Month to 18 Months |
| Sex | All |
| Enrollment | 10 (estimated) |
| Sponsor | Johns Hopkins University Academic / other |
| Locations | 1 site (Baltimore, Maryland) |
| Status | Recruiting |
| Started | September 17, 2026 |
| Main results expected | July 2028 |
| Study ends | July 2028 |
| Registry record updated | October 2, 2026 |
| Trial ID | NCT07778810 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are infants aged 1–18 months with clinical and imaging evidence of Sturge-Weber syndrome who have not yet had seizures.
✕Probably not a fit
Patients with significant liver disease, uncontrolled conditions, or other exclusion criteria may not benefit and could be at risk from the treatment.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This is a phase 2, open-label dose-escalation study in presymptomatic infants with Sturge-Weber syndrome to assess safety and tolerability of Epidiolex. Participants start at 2.5 mg/kg/day twice daily and escalate to up to 20 mg/kg/day while monitored with neurologic exams, blood tests, EEGs, and developmental assessments. The trial enrolls up to 10 babies, with an initial cohort of 6, and includes planned interim analysis after the first cohort. Neurologic and imaging data, plus pharmacokinetics, will inform whether treatment can delay seizure onset and support better neurological outcomes.
Why it matters
Potential benefit: If successful, presymptomatic Epidiolex could delay seizures and improve long-term neurological outcomes for infants with SWS.
How similar studies have gone: Previous open-label experience in this population suggested good tolerability of Epidiolex; efficacy in delaying seizures has not yet been established. This approach is exploratory and relatively novel for SWS.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * 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. Exclusion Criteria: 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. * 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.
Where it's running
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Kennedy Krieger Institute
Baltimore, Maryland, United States Recruiting
Study contacts
- Principal investigator: Ryan Felling, MD, PhD — Johns Hopkins School of Medicine
- Study coordinator: Anne Comi, MD
- Email: comi@kennedykrieger.org
- Phone: 443-923-9172
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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Tailored neuropsychological profile in Sturge-Weber Syndrome
This observational study tests the neuropsychological profile of people with Sturge-Weber Syndrome to understand cognitive strengths and weaknesses across ages.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.