Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber Syndrome
ConditionSturge - Weber Syndrome (SWS)
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age1-18
SponsorJohns Hopkins University
About this trial
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
Qualifiers
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.
Disqualifiers
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
Trial design
Treatments tested in this trial
- Epidiolex 100 mg/mL Oral Solution
Treatment groups
10 Participants
are divided into 1 treatment groupSponsors and collaborators
Johns Hopkins University
Lead sponsor
Jazz Pharmaceuticals
Collaborator
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Collaborator
Source ClinicalTrials.gov