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| Name | Class |
|---|---|
| Jazz Pharmaceuticals | INDUSTRY |
| Faneca 66 Foundation | OTHER |
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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).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cannabidiol/ Epidiolex | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Cannabidiol | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| List Sorting Working Memory Test | Data on cognitive function was collected using the List Sorting Working Memory Test from the NIH Toolbox. Data was collected on working memory performance, which was transformed into a t-score from 0 to 100 where a higher t-score indicates better performance. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and after 6 months on study drug. | Baseline, Follow-up (6 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Picture Vocabulary Test | Data on cognitive function was collected using the Picture Vocabulary subtest from the NIH Toolbox. Single words are presented via an audio file, paired simultaneously with 4 screen images of objects, actions, and/or depictions of concepts. The task is to pick the picture that matches the spoken word. Performance on the task was transformed into a t-score from 0 to 100 where a higher t-score indicates better performance. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and after 6 months on study drug. |
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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:
Exclusion Criteria:
Patients with any severe and/or uncontrolled medical conditions at randomization such as:
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.
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| Name | Affiliation | Role |
|---|---|---|
| Anne M Comi, MD | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
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| Label | URL |
|---|---|
| Hunter Nelson Sturge-Weber Center Website | View source |
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After consent, subject 10 was not enrolled as they did not meet the inclusion criteria of remaining at a stable does of anti-epileptic drugs 4 weeks prior to enrollment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cannabidiol/ Epidiolex | 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. Cannabidiol: 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Cannabidiol | Subjects took oral cannabidiol for 6 months (doses ranged from 5 mg/kg/day to 20 mg/kg/day). Neuropsychological, psychiatrics and motor assessments were administered at baseline and six months follow-up. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | List Sorting Working Memory Test | Data on cognitive function was collected using the List Sorting Working Memory Test from the NIH Toolbox. Data was collected on working memory performance, which was transformed into a t-score from 0 to 100 where a higher t-score indicates better performance. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and after 6 months on study drug. | Subject 1 was unable to complete this assessment due to lack of understanding of the instructions. Subject 10 was unable to complete this assessment due to a system error, where the fully adjusted score was not obtained. Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | T-score | Baseline, Follow-up (6 months) |
|
6 months
Serious adverse events include those events that: result in death; are life-threatening; require hospitalization or prolongation of existing hospitalization; create persistent, significant disability, or birth defects.
Expected adverse events are those that are identified in the research protocol as having been previously associated with participation in the study. All reported adverse events will be classified using the Common Terminology Criteria for Adverse Events Version 4.0.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cannabidiol/ Epidiolex | 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. Cannabidiol: 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. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Darkening of port-wine birthmark | Skin and subcutaneous tissue disorders | Systematic Assessment |
Due to the COVID-19 , visits transitioned from an in-person setting to a remote setting. Four subjects had baseline testing conducted in person and follow-up virtually, and 5 subjects had both baseline and follow-up testing virtually. The differences in testing environment and the context of the pandemic may have affected results. Also, due to variability in subject age and the small number of subjects, some participants were outside of assessment age norms.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Anne Comi | Kenndey Krieger Institute | 443-923-9127 | comi@kennedykrieger.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jun 29, 2021 | Jun 9, 2022 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D013341 | Sturge-Weber Syndrome |
| D060825 | Cognitive Dysfunction |
| ID | Term |
|---|---|
| D006391 | Hemangioma |
| D009383 | Neoplasms, Vascular Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D002185 | Cannabidiol |
| ID | Term |
|---|---|
| D002186 | Cannabinoids |
| D013729 | Terpenes |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
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|
| Baseline, Follow-up (6 months) |
| Seizure Frequency | Change in seizure frequency by seizure score at pre-treatment baseline and after six months. The seizure score is taken from the Sturge-Weber Neuroscore on a scale of 0 to 4 where 0=none, 1=1+. but controlled, 2=Breakthrough, 3=monthly, 4=Weekly+. Higher scores indicate worse outcome. | Baseline, Follow-up (6 months) |
| Migraine Severity | Data on migraine severity will be collected using patient responses to questions on a standard six-point scale. Data will be collected on the frequency of an event (e.g. feelings of frustration, performance of daily activities) which will be transformed into a score from 0 to 100 where higher scores indicate a less migraine severity and better outcomes. Data will be collected at baseline and after 6 months on study drug. | Baseline, Follow-up (6 months) |
| Modified House Classification Scores | Data on motor function was collected using a Modified House Classification. Data was collected on the ability to complete a task with the subject's non-dominant hand which was transformed into a score from 1 to 8 and 0 to 32 where higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Baseline, Follow-up (6 months) |
| Erhardt Developmental Prehension Assessment Scores | Data on motor function was collected using the Erhardt Developmental Prehension assessment. Data was collected on the ability to complete a task with each hand, which was scored as age equivalence of task performance (in months). Higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Baseline, Follow-up (6 months) |
| Pediatric Evaluation of Disability Inventory Computer Adapted Test | Data on motor function was collected using the Pediatric Evaluation of Disability Inventory Computer Adapted Test. Data was collected on the subject's ability to complete tasks involved in daily activities, mobility, and social/ cognitive activities. The data was transformed into scaled scores ranging from 20 to 80, where higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Baseline, Follow-up (6 months) |
| ABILHAND Questionnaire | Data on motor function was collected using the ABILHAND questionnaire, a measure of manual ability for adults with upper limb impairments. Data was collected on the subject's ability to complete daily activities that involve the upper limbs. Score was collected as a patient measure with scores ranging from -10 to 10. Higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Baseline, Follow-up (6 months) |
| Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV) | Data on cognitive function was collected using selected subtests, from either the Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or the Wechsler Adult Intelligence Scale (WAIS-IV). For the WISC-V and WAIS-IV subtests selected, scaled scores were derived from the normative data which account for age and demographic information. The selected WISC-V/WAIS-IV subtests were as follows: Digit Span, Symbol Search, Coding and Processing Speed; the first three subtests were scored from 0-10, the fourth subtest from 0-100, and for all subtests higher score is better. The selected subtests, from the WISC-V and the WAIS-IV, were combined to increase statistical power. Statistically rare changes in individual test scores were determined using a reliable change index methodology based upon normative information for these assessments. | Baseline, Follow-up (6 months) |
| Pediatric Neurological Quality of Life (Neuro-QoL) | Data on cognitive function was collected using Neurological Quality of Life scales from the NIH Toolbox. Data on frequency of an event (e.g. forgetting schoolwork) was collected and transformed into a t-score from 0 to 100 where higher t-scores indicate worse anger, worse anxiety, worse pain, better social relationships, worse stigma, worse depression, better cognitive function, and worse fatigue. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and 6 months on study drug. | Baseline, Follow-up (6 months) |
| Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) | Data on executive function was collected using the Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2). Data on the frequency of an event (e.g. becomes upset too easily) was collected and transformed into a t-score. The following BRIEF-2 subtests were evaluated: Behavioral Regulation Index, Emotional Regulation Index, Cognitive Regulation Index, and Global Executive Composite. For each BRIEF-2 subtest, t-scores range from 0 to 100; a score of 50 indicates the population mean with a standard deviation of 10. For all BASC-3 subtests, higher scores are worse outcome. | Baseline, Follow-up (6 months) |
| Social Responsiveness Scale, Second Edition (SRS-2) | Data on social function was collected using the Social Responsiveness Scale-Second Edition (SRS-2). Data on a child's ability to engage in emotionally appropriate reciprocal social interactions in naturalistic settings was collected and transformed into a t-score from 0 to 100 where higher scores indicate greater impairment in social function. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and 6 months on study drug. | Baseline, Follow-up (6 months) |
| Behavioral Assessment System for Children, Third Edition (BASC-3) | Data on behavioral function was collected using the Behavioral Assessment System for Children, Third Edition (BASC-3). Data on the frequency of a behavior (e.g. avoids eye contact) was collected and transformed into a t-score. Subscales for the BASC-3 scored were: External Problems Composite, the Internal Problems Composite, the Behavioral Symptoms Index, and the Adaptive Skills Composite. For the first three subscales, lower t-score indicates better outcome; for the fourth, a lower t-score indicates worse outcome. T-scores for all the BASC-3 subscales range from 0 to 100, and a t-score of 50 indicates the population mean with a standard deviation of 10. | Baseline, Follow-up (6 months) |
| Screen for Child Anxiety Related Disorders (SCARED) | Data on anxiety was collected using the Screen for Child Anxiety Related Disorders (SCARED). Data on the truthfulness of a statement (e.g. I am nervous) was collected and transformed into a score from 0 to 82 where higher scores indicate greater anxiety. Data was collected at baseline and 6 months on study drug. | Baseline, Follow-up (6 months) |
| Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55) | Data on quality of life was collected using the Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55). Data on the frequency of an event (e.g. had trouble concentrating on a task) was collected and transformed into a score from 0 to 100 where higher scores reflect better quality of life. Data was collected at baseline and 6 months on study drug. | Baseline, Follow-up (6 months) |
| Safety of Epidiolex | Safety of Epidiolex was measured by the number of adverse events and serious adverse events that result from study drug. | Baseline, Follow-up (6 months) |
| Neuroscore | Data on neurological function was collected using the Neuroscore. Data on frequency of seizures, extent of hemiparesis, assessment of visual field cut, and degree of cognitive functioning was transformed into a score from 0 to 15 where higher scores indicate worse neurologic function. Data was collected at baseline and 6 months on study drug. | Baseline, Follow-up (6 months) |
| Port-wine Birthmark Score | Data on facial port-wine birthmarks was collected using the Port-wine Birthmark Score. Data on percent of face covered, thickness of birthmark, and darkness of birthmark color was collected and transformed into a score from 0 to 43 where higher scores indicate greater severity and greater surface area involved. Data was collected at baseline and 6 months on study drug. | Baseline, Follow-up (6 months) |
| Adult Neurological Quality of Life (Neuro-QoL) | Data on cognitive function was collected using Neurological Quality of Life scales from the NIH Toolbox. Data on frequency of an event was collected and transformed into a t-score from 0 to 100. Higher t-scores indicate better communication, better ability to participate in social activity, worse anxiety, worse depression, worse emotional and behavioral dyscontrol, worse fatigue, better positive affect, worse sleep disturbance, better lower and upper extremity functions, worse stigma, better satisfaction with social roles, and better cognitive function. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and 6 months on study drug. | Baseline, Follow-up (6 months) |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Brain Involvement | Count of Participants | Participants |
|
| Skin Involvement | Count of Participants | Participants |
|
| Eye Involvement | Count of Participants | Participants |
|
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.
Cannabidiol: 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.
|
|
| Secondary | Picture Vocabulary Test | Data on cognitive function was collected using the Picture Vocabulary subtest from the NIH Toolbox. Single words are presented via an audio file, paired simultaneously with 4 screen images of objects, actions, and/or depictions of concepts. The task is to pick the picture that matches the spoken word. Performance on the task was transformed into a t-score from 0 to 100 where a higher t-score indicates better performance. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and after 6 months on study drug. | Subject 10 did not complete this assessment due to a technical issue where the fully adjusted score was not calculated. Subject 9 was withdrawn from the study prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | T-score | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Seizure Frequency | Change in seizure frequency by seizure score at pre-treatment baseline and after six months. The seizure score is taken from the Sturge-Weber Neuroscore on a scale of 0 to 4 where 0=none, 1=1+. but controlled, 2=Breakthrough, 3=monthly, 4=Weekly+. Higher scores indicate worse outcome. | Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Migraine Severity | Data on migraine severity will be collected using patient responses to questions on a standard six-point scale. Data will be collected on the frequency of an event (e.g. feelings of frustration, performance of daily activities) which will be transformed into a score from 0 to 100 where higher scores indicate a less migraine severity and better outcomes. Data will be collected at baseline and after 6 months on study drug. | Subject 7 misplaced this questionnaire and was not included in analysis. Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Modified House Classification Scores | Data on motor function was collected using a Modified House Classification. Data was collected on the ability to complete a task with the subject's non-dominant hand which was transformed into a score from 1 to 8 and 0 to 32 where higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Erhardt Developmental Prehension Assessment Scores | Data on motor function was collected using the Erhardt Developmental Prehension assessment. Data was collected on the ability to complete a task with each hand, which was scored as age equivalence of task performance (in months). Higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | Months | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Pediatric Evaluation of Disability Inventory Computer Adapted Test | Data on motor function was collected using the Pediatric Evaluation of Disability Inventory Computer Adapted Test. Data was collected on the subject's ability to complete tasks involved in daily activities, mobility, and social/ cognitive activities. The data was transformed into scaled scores ranging from 20 to 80, where higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | ABILHAND Questionnaire | Data on motor function was collected using the ABILHAND questionnaire, a measure of manual ability for adults with upper limb impairments. Data was collected on the subject's ability to complete daily activities that involve the upper limbs. Score was collected as a patient measure with scores ranging from -10 to 10. Higher scores indicate better motor function. Data was collected at baseline and after 6 months on study drug. | Subject 7 misplaced this questionnaire and was not included in analysis. Subject 9 was withdrawn prior to the 6 month follow-up visit due to persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV) | Data on cognitive function was collected using selected subtests, from either the Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or the Wechsler Adult Intelligence Scale (WAIS-IV). For the WISC-V and WAIS-IV subtests selected, scaled scores were derived from the normative data which account for age and demographic information. The selected WISC-V/WAIS-IV subtests were as follows: Digit Span, Symbol Search, Coding and Processing Speed; the first three subtests were scored from 0-10, the fourth subtest from 0-100, and for all subtests higher score is better. The selected subtests, from the WISC-V and the WAIS-IV, were combined to increase statistical power. Statistically rare changes in individual test scores were determined using a reliable change index methodology based upon normative information for these assessments. | Subjects 1 and 10 did not complete this questionnaire as they were too young. Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Pediatric Neurological Quality of Life (Neuro-QoL) | Data on cognitive function was collected using Neurological Quality of Life scales from the NIH Toolbox. Data on frequency of an event (e.g. forgetting schoolwork) was collected and transformed into a t-score from 0 to 100 where higher t-scores indicate worse anger, worse anxiety, worse pain, better social relationships, worse stigma, worse depression, better cognitive function, and worse fatigue. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and 6 months on study drug. | Subjects below the age of 18 completed the pediatric version of this questionnaire and were included in analysis. | Posted | Mean | Standard Deviation | T-score | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) | Data on executive function was collected using the Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2). Data on the frequency of an event (e.g. becomes upset too easily) was collected and transformed into a t-score. The following BRIEF-2 subtests were evaluated: Behavioral Regulation Index, Emotional Regulation Index, Cognitive Regulation Index, and Global Executive Composite. For each BRIEF-2 subtest, t-scores range from 0 to 100; a score of 50 indicates the population mean with a standard deviation of 10. For all BASC-3 subtests, higher scores are worse outcome. | Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | T-score | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Social Responsiveness Scale, Second Edition (SRS-2) | Data on social function was collected using the Social Responsiveness Scale-Second Edition (SRS-2). Data on a child's ability to engage in emotionally appropriate reciprocal social interactions in naturalistic settings was collected and transformed into a t-score from 0 to 100 where higher scores indicate greater impairment in social function. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and 6 months on study drug. | Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | T-score | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Behavioral Assessment System for Children, Third Edition (BASC-3) | Data on behavioral function was collected using the Behavioral Assessment System for Children, Third Edition (BASC-3). Data on the frequency of a behavior (e.g. avoids eye contact) was collected and transformed into a t-score. Subscales for the BASC-3 scored were: External Problems Composite, the Internal Problems Composite, the Behavioral Symptoms Index, and the Adaptive Skills Composite. For the first three subscales, lower t-score indicates better outcome; for the fourth, a lower t-score indicates worse outcome. T-scores for all the BASC-3 subscales range from 0 to 100, and a t-score of 50 indicates the population mean with a standard deviation of 10. | Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | T-score | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Screen for Child Anxiety Related Disorders (SCARED) | Data on anxiety was collected using the Screen for Child Anxiety Related Disorders (SCARED). Data on the truthfulness of a statement (e.g. I am nervous) was collected and transformed into a score from 0 to 82 where higher scores indicate greater anxiety. Data was collected at baseline and 6 months on study drug. | Subject 7 misplaced this assessment and was not included in analysis. Subject 9 was withdrawn from the study prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55) | Data on quality of life was collected using the Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55). Data on the frequency of an event (e.g. had trouble concentrating on a task) was collected and transformed into a score from 0 to 100 where higher scores reflect better quality of life. Data was collected at baseline and 6 months on study drug. | Subject 7 misplaced this assessment and was not included in analysis. Subject 9 was withdrawn prior to the 6 month follow-up appointment due to a persistent side effect and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Safety of Epidiolex | Safety of Epidiolex was measured by the number of adverse events and serious adverse events that result from study drug. | Posted | Number | Events | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Neuroscore | Data on neurological function was collected using the Neuroscore. Data on frequency of seizures, extent of hemiparesis, assessment of visual field cut, and degree of cognitive functioning was transformed into a score from 0 to 15 where higher scores indicate worse neurologic function. Data was collected at baseline and 6 months on study drug. | Subject 9 was withdrawn prior to the 6 month follow-up visit and not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Port-wine Birthmark Score | Data on facial port-wine birthmarks was collected using the Port-wine Birthmark Score. Data on percent of face covered, thickness of birthmark, and darkness of birthmark color was collected and transformed into a score from 0 to 43 where higher scores indicate greater severity and greater surface area involved. Data was collected at baseline and 6 months on study drug. | Subject 9 was withdrawn prior to the 6 month follow-up appointment and was not included in analysis. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Follow-up (6 months) |
|
|
|
| Secondary | Adult Neurological Quality of Life (Neuro-QoL) | Data on cognitive function was collected using Neurological Quality of Life scales from the NIH Toolbox. Data on frequency of an event was collected and transformed into a t-score from 0 to 100. Higher t-scores indicate better communication, better ability to participate in social activity, worse anxiety, worse depression, worse emotional and behavioral dyscontrol, worse fatigue, better positive affect, worse sleep disturbance, better lower and upper extremity functions, worse stigma, better satisfaction with social roles, and better cognitive function. T-score of 50 indicates the population mean with a standard deviation of 10. Data was collected at baseline and 6 months on study drug. | Subjects above the age of 18 completed the pediatric version of this questionnaire and were included in analysis. | Posted | Mean | Standard Deviation | T-score | Baseline, Follow-up (6 months) |
|
|
|
| 0 |
| 10 |
| 0 |
| 10 |
| 7 |
| 10 |
| Decreased appetite | Metabolism and nutrition disorders | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Dizziness | Ear and labyrinth disorders | Systematic Assessment |
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| Fatigue | Metabolism and nutrition disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Increased alanine aminotransferase | Hepatobiliary disorders | Systematic Assessment |
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| Increased aspartate aminotransferase | Hepatobiliary disorders | Systematic Assessment |
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| Irritable mood | Psychiatric disorders | Systematic Assessment |
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| Jitteriness | Psychiatric disorders | Systematic Assessment |
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| Mouth sores | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | Systematic Assessment |
|
Not provided
Not provided
| D020752 |
| Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D000798 | Angiomatosis |
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
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| Follow-up Mean Total Number of Items Successfully Completed |
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| Follow-up Mean Non-Dominant Hand Total Score |
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| Follow-Up Mean Score for Mobility |
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| Baseline Mean Score for Responsibility |
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| Follow-Up Mean Score for Responsibility |
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| Baseline Mean Score for Social Cognitive |
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| Follow-up Mean Score for Social Cognitive |
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| Follow-up Mean Symbol Search Score |
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| Baseline Mean Coding Score |
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| Follow-up Mean Coding Score |
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| Baseline Mean Processing Speed Score |
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| Follow-up Mean Processing Speed Score |
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| Follow-up Mean Pediatric Score for Anxiety |
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| Baseline Mean Pediatric Score for Pain |
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| Follow-up Mean Pediatric Score for Pain |
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| Baseline Mean Pediatric Score for Social Relations |
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| Follow-up Mean Pediatric Score for Social Relations |
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| Baseline Mean Pediatric Score for Stigma |
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| Follow-up Mean Pediatric Score for Stigma |
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| Baseline Mean Pediatric Score for Depression |
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| Follow-up Mean Pediatric Score for Depression |
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| Baseline Mean Pediatric Score for Cognitive Function |
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| Follow-up Mean Pediatric Score for Cognitive Function |
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| Baseline Mean Pediatric Score for Fatigue |
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| Follow-up Mean Pediatric Score for Fatigue |
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| Follow-up Mean for Emotional Regulation Index |
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| Baseline Mean for Cognitive Regulation Index |
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| Follow-up Mean for Cognitive Regulation Index |
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| Baseline Mean for Global Executive Composite |
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| Follow-up Mean for Global Executive Composite |
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| Follow-Up Mean Internal Problems Composite |
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| Baseline Mean Behavioral Symptoms Index |
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| Follow-up Mean Behavioral Symptoms Index |
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| Baseline Mean Adaptive Skills Composite |
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| Follow-Up Mean Adaptive Skills Composite |
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| Title | Measurements |
|---|---|
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| Follow-Up Mean Hemiparesis Score |
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| Baseline Mean Visual Field Cut Score |
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| Follow-Up Mean Visual Field Cut Score |
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| Baseline Mean Cognitive Function Score |
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| Follow-up Mean Cognitive Function Score |
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| Baseline Mean Composite Score |
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| Follow-up Mean Composite Score |
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| Follow-Up Mean Adult Score for Depression |
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| Baseline Mean Adult Score for Fatigue |
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| Follow-Up Mean Adult Score for Fatigue |
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| Baseline Mean Adult Score for Upper Extremity Function |
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| Follow-Up Mean Adult Score for Upper Extremity Function |
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| Baseline Mean Adult Score for Lower Extremity Function |
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| Follow-Up Mean Adult Score for Lower Extremity Function |
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| Baseline Mean Adult Score for Cognitive Function |
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| Follow-Up Mean Adult Score for Cognitive Function |
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| Baseline Mean Adult Score for Emotional and Behavioral Dyscontrol |
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| Follow-Up Mean Adult Score for Emotional and Behavioral Dyscontrol |
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| Baseline Mean Adult Score for Well-being |
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| Follow-Up Mean Adult Score for Well-being |
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| Baseline Mean Adult Score for Sleep Disturbances |
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| Follow-Up Mean Adult Score for Sleep Disturbances |
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| Baseline Mean Adult Score for Participation in Social Activities |
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| Follow-Up Mean Adult Score for Participation in Social Activities |
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| Baseline Mean Adult Score for Satisfaction in Social Activities |
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| Follow-Up Mean Adult Score for Satisfaction in Social Activities |
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| Baseline Mean Adult Score for Stigma |
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| Follow-Up Mean Adult Score for Stigma |
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