NCT07778810 Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber Syndrome
| NCT ID | NCT07778810 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Johns Hopkins University |
| Condition | Sturge - Weber Syndrome (SWS) |
| Study Type | INTERVENTIONAL |
| Enrollment | 10 participants |
| Start Date | 2026-09-17 |
| Primary Completion | 2028-07 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 10 participants in total. It began in 2026-09-17 with a primary completion date of 2028-07.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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. This trial will evaluate whether Epidiolex in presymptomatic Sturge-Weber patients may delay the onset of seizures and improve neurological outcome.
Eligibility 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.
Contact & Investigator
Ryan Felling, MD, PhD
PRINCIPAL INVESTIGATOR
Johns Hopkins School of Medicine
Frequently Asked Questions
Who can join the NCT07778810 clinical trial?
This trial is open to participants of all sexes, aged 1 Month or older, up to 18 Months, studying Sturge - Weber Syndrome (SWS). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07778810 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT07778810 currently recruiting?
Yes, NCT07778810 is actively recruiting participants. Contact the research team at comi@kennedykrieger.org for enrollment information.
Where is the NCT07778810 trial being conducted?
This trial is being conducted at Baltimore, United States.
Who is sponsoring the NCT07778810 clinical trial?
NCT07778810 is sponsored by Johns Hopkins University. The principal investigator is Ryan Felling, MD, PhD at Johns Hopkins School of Medicine. The trial plans to enroll 10 participants.