Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber Syndrome

NCT07778810 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: IRB00326943
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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)

Keywords

Presymptomatic; Open-label; Cannabidiol

Outcome Measures

Primary Outcomes (2)

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

  6 months

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

  6 months

Secondary Outcomes (5)

• Sturge-Weber Syndrome Neuroscore (SWS-NRS)

  baseline, week 5, week 12, and week 24

• Milestone checklist

  baseline, week 5, week 12, and week 24

• Modified Early Motor Questionnaire (EMQ)

  Baseline, week 24

• Modified Erhardt Developmental Prehension Assessment (EDPA)

  Baseline, week 24

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

  Baseline, week 24

Study Arms (1)

Presymptomatic Epidiolex Treatment

EXPERIMENTAL

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.

Drug: Epidiolex 100 mg/mL Oral Solution

Interventions

Epidiolex 100 mg/mL Oral Solution DRUG

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.

Also known as: Cannabidiol, CBD

Presymptomatic Epidiolex Treatment

Eligibility Criteria

• Age: 1 Month - 18 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Johns Hopkins University: lead
• Jazz Pharmaceuticals: collaborator
• Hugo W. Moser Research Institute at Kennedy Krieger, Inc.: collaborator

Study Sites (1)

Kennedy Krieger Institute

Baltimore, Maryland, 21205, United States

Location

MeSH Terms

Conditions

Brain Stem Infarctions

Interventions

Cannabidiol; Solutions

Condition Hierarchy (Ancestors)

Brain Infarction; Brain Ischemia; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Stroke; Vascular Diseases; Cardiovascular Diseases; Infarction; Ischemia; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Necrosis

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals; Pharmaceutical Preparations

Study Officials

• Ryan Felling, MD, PhD

  Johns Hopkins School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Anne Comi, MD

CONTACT

443-923-9172; comi@kennedykrieger.org

Brenna Keam, BS

CONTACT

443-923-9127; keam@kennedykrieger.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Open-label

Study Record Dates

• First Submitted: August 18, 2026
• First Posted: August 21, 2026
• Study Start: August 1, 2026
• Primary Completion (Estimated): July 1, 2028
• Study Completion (Estimated): July 1, 2028
• Last Updated: August 26, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: SAP
• Time Frame: July 1, 2028 until July 1, 2029
• Access Criteria: Supplemental data with paper when published. Other interested parties can reach out to comi@kennedykrieger.org to discuss other arrangements.

Locations

US (1)