Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome

Anne Comi, MD

Status and phase: Completed; Phase 2

Conditions: Sturge-Weber Syndrome

Treatments: Drug: Cannabidiol

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04447846

IRB00204427

Details and patient eligibility

About

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).

Full description

The investigators hope to gain an understanding of the utility of pharmaceutical grade CBD used for the treatment of cognitive impairments in SWS in this open-label study. Anecdotal evidence from a phase I trial investigating the use of CBD for medically refractory seizures suggests CBD may also have a beneficial effect on cognition, mood, and behavior. The investigators hypothesize that CBD/ Epidiolex will improve SWS brain function resulting in improved cognitive function, social interactions, mood, motor function and behavior, as well as reduced migraines. This is an open-label prospective oral drug trial of Epidiolex in 10 subjects. Assessments will be done at baseline and repeated after 6 months on study drug.

Enrollment: 11 patients

Sex: All

Ages: 3 to 50 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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11 participants in 1 patient group

Cannabidiol/ Epidiolex

Experimental group

Description:

All subjects will receive the experimental Epidiolex (cannabidiol) oral solution to be taken at home twice a day, and will be treated on an outpatient basis. The drug will be taken for 24 weeks unless the subject chooses to participate in the extension phase of the study, in which case the subject will continue to receive the drug for one additional year or until the drug is approved for clinical use for the treatment of cognitive impairments in patients with Sturge-Weber syndrome.

Treatment:

Drug: Cannabidiol