Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome
Condition: Sturge-Weber Syndrome
MeSH terms: Sturge-Weber Syndrome; Brain Stem Infarctions; Klippel-Trenaunay-Weber Syndrome; Syndrome
Intervention: Cannabidiol
Intervention description: Initiation of treatment will begin with 5 mg/kg/day given in two divided doses. The dose will be increased by 5 mg/kg/day after seven days and then by 5 mg/kg/day every seven days up to a maximum dose of 20 mg/kg/day given.
Intervention MeSH terms: Cannabidiol
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 11.0
Sex: All
Minimum age: 3 Years
Maximum age: 50 Years
Healthy volunteers: No
Keywords: Sturge-Weber Syndrome; SWS; Cognitive impairments

Summary:

      The purpose of this study is to better understand the utility of cannabidiol (CBD/ Epidiolex)
      for improving the treatment of cognitive impairments in Sturge-Weber syndrome (SWS).
    

Eligibility criteria:

        Inclusion Criteria: Participants with Sturge-Weber syndrome brain involvement as defined on
        neuroimaging (n=10 subjects, male and female, ages 3 to 50 years of age) and the following:

          -  Cognitive impairment defined as a cognitive neuroscore greater than or equal to 2 at
             screening.

          -  Anti-epileptic, mood or behavioral drugs (if on) at stable doses for a minimum of 4
             weeks prior to enrollment.

          -  If present, VNS must be on stable setting for a minimum of 3 months prior to
             enrollment.

          -  If on ketogenic or Atkins diet, must be on stable ratio for a minimum of 3 months
             prior to enrollment.

          -  Previous subjects who fail at any point to meet continuation criteria and withdraw
             early may be considered for re-enrollment under new subject ID as long as the above
             inclusion criteria are met. The determination of whether to re-enroll will be made by
             the PI and sponsor on a case-by-case basis. Re-enrollment can occur no earlier than 4
             weeks after the final, post-weaning follow-up visit under the old subject ID.

          -  Written informed consent obtained from the patient or the patient's legal
             representative must be obtained prior to beginning treatment.

        Exclusion Criteria:

          -  Patients with any severe and/or uncontrolled medical conditions at randomization such
             as:

               1. Liver disease such as cirrhosis, decompensated liver disease, and chronic
                  hepatitis (i.e. quantifiable HBV-DNA and/or positive HbsAg, quantifiable HCV-RNA)

               2. Uncontrolled diabetes as defined by fasting serum glucose greater than 1.5

               3. Active (acute or chronic) or uncontrolled severe infections

               4. Active, bleeding diathesis

          -  Patients who have 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.

          -  Patients who start or discontinue a seizure, mood or behavioral medication in the 4
             weeks leading up to screening.

          -  Prior treatment with any investigational drug or use of any other cannabis product
             within the preceding 4 weeks prior to study entry.

          -  Patients with a history of non-compliance to medical regimens or who are considered
             potentially unreliable or will not be able to complete the entire study. This includes
             those in foster care, or those unable to keep follow-up appointments, maintain close
             contact with the Principal Investigator, or complete all necessary studies to maintain
             safety.

          -  Pregnant or nursing (lactating) women, where pregnancy is defined as the state of the
             female after conception and until the termination of gestation, confirmed by a
             positive hCG laboratory test.