Clinical trial NCT04447846: Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome
Official title: Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome: A Phase II Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Sturge-Weber Syndrome
Keywords: Sturge-Weber Syndrome
SWS
Cognitive impairments
Lead sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Sponsor class: OTHER
Collaborators: name: Jazz Pharmaceuticals
name: Faneca 66 Foundation
First posted: 2020-06-25
Enrollment: 11
Sex: ALL
Minimum age (years): 3
Maximum age (years): 50
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).

Detailed 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.

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.

Primary outcomes:
measure: List Sorting Working Memory Test
timeFrame: Baseline, Follow-up (6 months)
Secondary outcomes:
measure: Picture Vocabulary Test
timeFrame: Baseline, Follow-up (6 months)
measure: Seizure Frequency
timeFrame: Baseline, Follow-up (6 months)
measure: Migraine Severity
timeFrame: Baseline, Follow-up (6 months)
measure: Modified House Classification Scores
timeFrame: Baseline, Follow-up (6 months)
measure: Erhardt Developmental Prehension Assessment Scores
timeFrame: Baseline, Follow-up (6 months)
measure: Pediatric Evaluation of Disability Inventory Computer Adapted Test
timeFrame: Baseline, Follow-up (6 months)
measure: ABILHAND Questionnaire
timeFrame: Baseline, Follow-up (6 months)
measure: Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
timeFrame: Baseline, Follow-up (6 months)
measure: Pediatric Neurological Quality of Life (Neuro-QoL)
timeFrame: Baseline, Follow-up (6 months)
measure: Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
timeFrame: Baseline, Follow-up (6 months)
measure: Social Responsiveness Scale, Second Edition (SRS-2)
timeFrame: Baseline, Follow-up (6 months)
measure: Behavioral Assessment System for Children, Third Edition (BASC-3)
timeFrame: Baseline, Follow-up (6 months)
measure: Screen for Child Anxiety Related Disorders (SCARED)
timeFrame: Baseline, Follow-up (6 months)
measure: Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55)
timeFrame: Baseline, Follow-up (6 months)
measure: Safety of Epidiolex
timeFrame: Baseline, Follow-up (6 months)
measure: Neuroscore
timeFrame: Baseline, Follow-up (6 months)
measure: Port-wine Birthmark Score
timeFrame: Baseline, Follow-up (6 months)
measure: Adult Neurological Quality of Life (Neuro-QoL)
timeFrame: Baseline, Follow-up (6 months)
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
Study officials:
affiliation: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
name: Anne M Comi, MD
role: PRINCIPAL_INVESTIGATOR
References: