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Epidiolex Trial for Presymptomatic Treatment of Sturge-Weber Syndrome

ConditionSturge - Weber Syndrome (SWS)
Trial statusRecruiting
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

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

  • 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.

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: Presymptomatic Epidiolex TreatmentExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
6 months
2

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

Time frame
6 months

Secondary outcomes

1

Sturge-Weber Syndrome Neuroscore (SWS-NRS)

Neuroscore is a published SWS ordinal score that incorporates domains of visual field cut (0-2), hemiparesis (0-4), seizure frequency (0-4), and cognitive function (0-5). Total score is sum of 4 ratings with a total score from 0-15. Higher score worse impairment. This will be obtained at each study visit.

Time frame
baseline, week 5, week 12, and week 24
2

Milestone checklist

A publicly available milestone checklist from Pathways.org will be completed at every study visit. The checklist is personalized based on age. Does not use a numerical or point based scoring system. It uses a pass/fail or completed/not-completed checklist tracking format related to Motor, Sensory, Communication, and Feeding abilities.

Time frame
baseline, week 5, week 12, and week 24
3

Modified Early Motor Questionnaire (EMQ)

The Modified EMQ will be performed at baseline and at week 24. The EMQ evaluates abilities in three domains: Gross Motor skills, Fine Motor Skills, and Perception-Action Integration. It uses a 5-point scale including -2 = Sure that child does NOT show behavior, -1 = Child probably does NOT show behavior yet, 0 = Unsure whether child could do this or not, 1 = Child probably shows this behavior, and 2 = Sure that child shows this behavior and remember a particular instance. Minimum total score is -200. Maximum total score is 200. Higher score means higher certainty in the child's behaviors.

Time frame
Baseline, week 24
4

Modified Erhardt Developmental Prehension Assessment (EDPA)

The Modified EDPA will be performed at baseline and at week 24. The EDPA will evaluate hand skills and arm-hand movements at baseline and in response to the study drug. Both right and left hands are tested. Individual test components are scored by a well-established pattern (+), pattern not yet achieved (-), and temporary pattern to be replaced by a more mature pattern (++). Maximum score would be a well-established pattern (+) for all EDPA items. Higher score better, means a greater number of well-established hand skills and arm-hand movements.

Time frame
Baseline, week 24

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Johns Hopkins University

Lead sponsor

Jazz Pharmaceuticals

Collaborator

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Collaborator

Source ClinicalTrials.gov
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