Recruiting
Phase 2

Epidiolex

Sponsor:

Johns Hopkins University

Code:

NCT07778810

Conditions

Sturge - Weber Syndrome (SWS)

Eligibility Criteria

Sex: All

Age: 0 - 1

Healthy Volunteers: Not accepted

Interventions

Epidiolex 100 mg/mL Oral Solution

Study Details

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.

Conditions

Sturge - Weber Syndrome (SWS)

Study ID

NCT07778810

Start date

Sep, 2026

Status verified date

Sep, 2026

Completion date

Jul, 2028

Anticipated

Primary completion date

Jul, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 1

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

10 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Presymptomatic Epidiolex Treatment

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.

Interventions

Epidiolex 100 mg/mL Oral Solution

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.

Primary outcome measure

  • Safe completion as assessed by the number of participants with no significant side effects [ Time Frame: 6 months ]
  • Safe completion as assessed by the number of participants with no seizure onset [ Time Frame: 6 months ]

Central Contacts and Locations

Central contacts

Anne Comi, MD

443-923-9172comi@kennedykrieger.org

Brenna Keam, BS

443-923-9127keam@kennedykrieger.org

Locations

Kennedy Krieger Institute

Recruiting

Baltimore, Maryland, United States, 21205

Contacts

Anne Comi, MD

443-923-9172comi@kennedykrieger.org

Brenna Keam, BS

443-923-9127keam@kennedykrieger.org

Principal Investigator:

Ryan Felling, MD, PhD

More Information

Sponsor

Johns Hopkins University

Last update posted

Sep 25, 2026

Last verified

Sep, 2026

Keywords

  • Presymptomatic
  • Open-label
  • Cannabidiol

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-26. This information was provided to ClinicalTrials.gov by Johns Hopkins University on 2026-09-25. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.