Trial Outcomes & Findings for Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome (NCT NCT04447846)
NCT ID: NCT04447846
Last Updated: 2023-05-25
Results Overview
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.
COMPLETED
PHASE2
11 participants
Baseline, Follow-up (6 months)
2023-05-25
Participant Flow
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.
Participant milestones
| Measure |
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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|---|---|
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Overall Study
STARTED
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10
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Overall Study
COMPLETED
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9
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Overall Study
NOT COMPLETED
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1
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Reasons for withdrawal
| Measure |
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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|---|---|
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Overall Study
Withdrew due to moderate side effects (fatigue)
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1
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Baseline Characteristics
Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome
Baseline characteristics by cohort
| Measure |
Cannabidiol
n=10 Participants
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.
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|---|---|
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Age, Continuous
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13.83 years
STANDARD_DEVIATION 9.67 • n=5 Participants
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Sex: Female, Male
Female
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6 Participants
n=5 Participants
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Sex: Female, Male
Male
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4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
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2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
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7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
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1 Participants
n=5 Participants
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|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
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2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
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1 Participants
n=5 Participants
|
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Region of Enrollment
United States
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10 participants
n=5 Participants
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Brain Involvement
Right-Sided Brain Involvement
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6 Participants
n=5 Participants
|
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Brain Involvement
Left-Sided Brain Involvement
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2 Participants
n=5 Participants
|
|
Brain Involvement
Bilateral Brain Involvement
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2 Participants
n=5 Participants
|
|
Skin Involvement
Right-Sided Skin Involvement
|
4 Participants
n=5 Participants
|
|
Skin Involvement
Left-Sided Skin Involvement
|
2 Participants
n=5 Participants
|
|
Skin Involvement
Bilateral Skin Involvement
|
4 Participants
n=5 Participants
|
|
Eye Involvement
Right-Sided Eye Involvement
|
4 Participants
n=5 Participants
|
|
Eye Involvement
Left-Sided Eye Involvement
|
2 Participants
n=5 Participants
|
|
Eye Involvement
Bilateral Eye Involvement
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1 Participants
n=5 Participants
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Eye Involvement
No Eye Involvement
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3 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: 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.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=7 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.
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|---|---|
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List Sorting Working Memory Test
Baseline List Sorting Working Memory Fully Adjusted Score
|
37.14 T-score
Standard Deviation 10.92
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List Sorting Working Memory Test
Follow-Up List Sorting Working Memory Fully Adjusted Score
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39.14 T-score
Standard Deviation 13.78
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: 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.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=8 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.
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|---|---|
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Picture Vocabulary Test
Baseline Mean of the Fully Adjusted Picture Vocabulary Score
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42.88 T-score
Standard Deviation 8.86
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Picture Vocabulary Test
Follow-up Mean of the Fully Adjusted Picture Vocabulary Score
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42.38 T-score
Standard Deviation 9.43
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
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|---|---|
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Seizure Frequency
Baseline Seizure Score from Sturge-Weber Neuroscore
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1.11 score on a scale
Standard Deviation 0.33
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Seizure Frequency
Follow-up Seizure Score from Sturge-Weber Neuroscore
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1.00 score on a scale
Standard Deviation 0.00
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: 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.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=8 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.
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|---|---|
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Migraine Severity
Baseline Mean Migraine-Quality of Life Score
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96.83 score on a scale
Standard Deviation 7.62
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Migraine Severity
Follow-up Mean Migraine-Quality of Life Score
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97.50 score on a scale
Standard Deviation 4.69
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
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|---|---|
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Modified House Classification Scores
Baseline Mean Highest Category in Which All Items were Successfully Completed
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5.11 score on a scale
Standard Deviation 0.93
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Modified House Classification Scores
Follow-up Mean Highest Category in Which All Items were Successfully Completed
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5.44 score on a scale
Standard Deviation 1.33
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Modified House Classification Scores
Baseline Mean Total Number of Items Successfully Completed
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26.67 score on a scale
Standard Deviation 4.12
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Modified House Classification Scores
Follow-up Mean Total Number of Items Successfully Completed
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27.44 score on a scale
Standard Deviation 4.75
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
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|---|---|
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Erhardt Developmental Prehension Assessment Scores
Baseline Mean Dominant Hand Total Score
|
54.22 Months
Standard Deviation 2.64
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Erhardt Developmental Prehension Assessment Scores
Follow-up Mean Dominant Hand Total Score
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53.56 Months
Standard Deviation 4.04
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Erhardt Developmental Prehension Assessment Scores
Baseline Mean Non-Dominant Hand Total Score
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51.22 Months
Standard Deviation 9.48
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Erhardt Developmental Prehension Assessment Scores
Follow-up Mean Non-Dominant Hand Total Score
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53.78 Months
Standard Deviation 4.15
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
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|---|---|
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Baseline Mean Score for Daily Activities
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58.67 score on a scale
Standard Deviation 7.76
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Follow-up Mean Score for Daily Activities
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60.00 score on a scale
Standard Deviation 6.50
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Baseline Mean Score for Mobility
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67.11 score on a scale
Standard Deviation 4.76
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Follow-Up Mean Score for Mobility
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68.56 score on a scale
Standard Deviation 4.07
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Baseline Mean Score for Responsibility
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52.44 score on a scale
Standard Deviation 11.67
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Follow-Up Mean Score for Responsibility
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53.89 score on a scale
Standard Deviation 11.10
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Baseline Mean Score for Social Cognitive
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66.89 score on a scale
Standard Deviation 5.35
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Pediatric Evaluation of Disability Inventory Computer Adapted Test
Follow-up Mean Score for Social Cognitive
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67.22 score on a scale
Standard Deviation 4.63
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: 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.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=8 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.
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|---|---|
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ABILHAND Questionnaire
Baseline Mean of the ABILHAND
|
1.81 score on a scale
Standard Deviation 1.98
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ABILHAND Questionnaire
Follow-up Mean of the ABILHAND
|
2.88 score on a scale
Standard Deviation 1.39
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: 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.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=7 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.
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|---|---|
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Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Baseline Mean Digit Span Score
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5.71 score on a scale
Standard Deviation 2.81
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Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Follow-up Mean Digit Span Score
|
6.43 score on a scale
Standard Deviation 2.07
|
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Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Baseline Mean Symbol Search Score
|
6.00 score on a scale
Standard Deviation 2.71
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|
Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Follow-up Mean Symbol Search Score
|
5.57 score on a scale
Standard Deviation 2.07
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Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Baseline Mean Coding Score
|
5.57 score on a scale
Standard Deviation 2.88
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|
Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Follow-up Mean Coding Score
|
4.71 score on a scale
Standard Deviation 2.22
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Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Baseline Mean Processing Speed Score
|
77.14 score on a scale
Standard Deviation 13.93
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|
Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)
Follow-up Mean Processing Speed Score
|
73.43 score on a scale
Standard Deviation 11.19
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SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subjects below the age of 18 completed the pediatric version of this questionnaire and were included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=6 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.
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|---|---|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Anxiety
|
41.07 T-score
Standard Deviation 5.74
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Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Anger
|
53.02 T-score
Standard Deviation 3.09
|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Anger
|
49.67 T-score
Standard Deviation 7.00
|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Anxiety
|
45.20 T-score
Standard Deviation 5.79
|
|
Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Pain
|
40.20 T-score
Standard Deviation 4.16
|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Pain
|
43.50 T-score
Standard Deviation 5.89
|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Social Relations
|
43.28 T-score
Standard Deviation 15.10
|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Social Relations
|
54.33 T-score
Standard Deviation 6.96
|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Stigma
|
42.45 T-score
Standard Deviation 6.42
|
|
Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Stigma
|
44.52 T-score
Standard Deviation 4.88
|
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Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Depression
|
44.07 T-score
Standard Deviation 6.29
|
|
Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Depression
|
44.62 T-score
Standard Deviation 8.58
|
|
Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Cognitive Function
|
49.27 T-score
Standard Deviation 3.36
|
|
Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Cognitive Function
|
51.33 T-score
Standard Deviation 6.44
|
|
Pediatric Neurological Quality of Life (Neuro-QoL)
Baseline Mean Pediatric Score for Fatigue
|
46.38 T-score
Standard Deviation 9.44
|
|
Pediatric Neurological Quality of Life (Neuro-QoL)
Follow-up Mean Pediatric Score for Fatigue
|
45.97 T-score
Standard Deviation 8.14
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
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|---|---|
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Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Baseline Mean for Behavioral Regulation Index
|
55.11 T-score
Standard Deviation 11.66
|
|
Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Follow-Up Mean for Behavioral Regulation Index
|
52.44 T-score
Standard Deviation 13.60
|
|
Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Baseline Mean for Emotional Regulation Index
|
60.22 T-score
Standard Deviation 10.24
|
|
Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Follow-up Mean for Emotional Regulation Index
|
51.22 T-score
Standard Deviation 12.29
|
|
Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Baseline Mean for Cognitive Regulation Index
|
54.11 T-score
Standard Deviation 7.06
|
|
Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Follow-up Mean for Cognitive Regulation Index
|
54.22 T-score
Standard Deviation 10.59
|
|
Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Baseline Mean for Global Executive Composite
|
56.78 T-score
Standard Deviation 8.17
|
|
Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2)
Follow-up Mean for Global Executive Composite
|
53.33 T-score
Standard Deviation 11.57
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
|
|---|---|
|
Social Responsiveness Scale, Second Edition (SRS-2)
Baseline Mean Total Score
|
54.22 T-score
Standard Deviation 7.71
|
|
Social Responsiveness Scale, Second Edition (SRS-2)
Follow-up Mean Total Score
|
49.22 T-score
Standard Deviation 7.09
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
|
|---|---|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Baseline Mean External Problems Composite
|
51.22 T-score
Standard Deviation 6.10
|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Follow-up Mean External Problems Composite
|
50.11 T-score
Standard Deviation 6.31
|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Baseline Mean Internal Problems Composite
|
52.22 T-score
Standard Deviation 5.81
|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Follow-Up Mean Internal Problems Composite
|
46.22 T-score
Standard Deviation 7.17
|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Baseline Mean Behavioral Symptoms Index
|
54.22 T-score
Standard Deviation 4.21
|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Follow-up Mean Behavioral Symptoms Index
|
50.11 T-score
Standard Deviation 8.19
|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Baseline Mean Adaptive Skills Composite
|
45.00 T-score
Standard Deviation 8.29
|
|
Behavioral Assessment System for Children, Third Edition (BASC-3)
Follow-Up Mean Adaptive Skills Composite
|
48.44 T-score
Standard Deviation 7.63
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: 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.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=8 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.
|
|---|---|
|
Screen for Child Anxiety Related Disorders (SCARED)
Baseline Mean of Total Score
|
14.86 score on a scale
Standard Deviation 14.49
|
|
Screen for Child Anxiety Related Disorders (SCARED)
Follow-up Mean of Total Score
|
11.57 score on a scale
Standard Deviation 11.16
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: 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.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=8 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.
|
|---|---|
|
Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55)
Baseline Mean Total Score
|
61.17 score on a scale
Standard Deviation 18.14
|
|
Quality of Life in Childhood Epilepsy Questionnaire (QOLCE-55)
Follow-up Mean Total Score
|
71.39 score on a scale
Standard Deviation 10.75
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Safety of Epidiolex was measured by the number of adverse events and serious adverse events that result from study drug.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=10 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.
|
|---|---|
|
Safety of Epidiolex
Number of Adverse Events that Resulted from the Study Drug
|
24 Events
|
|
Safety of Epidiolex
Number of Serious Adverse Events that Resulted from the Study Drug
|
0 Events
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up visit and not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
|
|---|---|
|
Neuroscore
Baseline Mean Seizure Score
|
1.11 score on a scale
Standard Deviation 0.33
|
|
Neuroscore
Follow-Up Mean Seizure Score
|
1.00 score on a scale
Standard Deviation 0.00
|
|
Neuroscore
Baseline Mean Hemiparesis Score
|
1.44 score on a scale
Standard Deviation 1.13
|
|
Neuroscore
Follow-Up Mean Hemiparesis Score
|
1.00 score on a scale
Standard Deviation 0.87
|
|
Neuroscore
Baseline Mean Visual Field Cut Score
|
0.11 score on a scale
Standard Deviation 0.33
|
|
Neuroscore
Follow-Up Mean Visual Field Cut Score
|
0.00 score on a scale
Standard Deviation 0.00
|
|
Neuroscore
Baseline Mean Cognitive Function Score
|
2.33 score on a scale
Standard Deviation 0.50
|
|
Neuroscore
Follow-up Mean Cognitive Function Score
|
1.44 score on a scale
Standard Deviation 0.53
|
|
Neuroscore
Baseline Mean Composite Score
|
5.00 score on a scale
Standard Deviation 1.32
|
|
Neuroscore
Follow-up Mean Composite Score
|
3.44 score on a scale
Standard Deviation 0.88
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subject 9 was withdrawn prior to the 6 month follow-up appointment and was not included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=9 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.
|
|---|---|
|
Port-wine Birthmark Score
Baseline Mean Total Score
|
8.56 score on a scale
Standard Deviation 3.09
|
|
Port-wine Birthmark Score
Follow-up Mean Total Score
|
8.11 score on a scale
Standard Deviation 3.18
|
SECONDARY outcome
Timeframe: Baseline, Follow-up (6 months)Population: Subjects above the age of 18 completed the pediatric version of this questionnaire and were included in analysis.
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.
Outcome measures
| Measure |
Cannabidiol/ Epidiolex
n=3 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.
|
|---|---|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Anxiety
|
46.77 T-score
Standard Deviation 13.33
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Anxiety
|
46.87 T-score
Standard Deviation 6.45
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Depression
|
42.70 T-score
Standard Deviation 10.05
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Depression
|
44.23 T-score
Standard Deviation 9.53
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Fatigue
|
45.23 T-score
Standard Deviation 9.53
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Fatigue
|
42.80 T-score
Standard Deviation 4.60
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Upper Extremity Function
|
48.97 T-score
Standard Deviation 8.37
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Upper Extremity Function
|
53.80 T-score
Standard Deviation 0.00
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Lower Extremity Function
|
58.60 T-score
Standard Deviation 0.00
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Lower Extremity Function
|
58.60 T-score
Standard Deviation 0.00
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Cognitive Function
|
47.03 T-score
Standard Deviation 15.52
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Cognitive Function
|
46.73 T-score
Standard Deviation 5.01
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Emotional and Behavioral Dyscontrol
|
51.10 T-score
Standard Deviation 14.97
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Emotional and Behavioral Dyscontrol
|
41.37 T-score
Standard Deviation 9.98
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Well-being
|
54.30 T-score
Standard Deviation 4.91
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Well-being
|
55.70 T-score
Standard Deviation 6.59
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Sleep Disturbances
|
47.03 T-score
Standard Deviation 7.49
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Sleep Disturbances
|
43.93 T-score
Standard Deviation 4.90
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Participation in Social Activities
|
53.00 T-score
Standard Deviation 7.41
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Participation in Social Activities
|
51.03 T-score
Standard Deviation 8.01
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Satisfaction in Social Activities
|
47.70 T-score
Standard Deviation 3.28
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Satisfaction in Social Activities
|
49.23 T-score
Standard Deviation 2.54
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Baseline Mean Adult Score for Stigma
|
53.90 T-score
Standard Deviation 8.80
|
|
Adult Neurological Quality of Life (Neuro-QoL)
Follow-Up Mean Adult Score for Stigma
|
56.47 T-score
Standard Deviation 6.25
|
Adverse Events
Cannabidiol/ Epidiolex
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cannabidiol/ Epidiolex
n=10 participants at risk
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.
|
|---|---|
|
Skin and subcutaneous tissue disorders
Darkening of port-wine birthmark
|
10.0%
1/10 • Number of events 1 • 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.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
10.0%
1/10 • Number of events 3 • 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.
|
|
Gastrointestinal disorders
Diarrhea
|
20.0%
2/10 • Number of events 2 • 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.
|
|
Ear and labyrinth disorders
Dizziness
|
20.0%
2/10 • Number of events 3 • 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.
|
|
Metabolism and nutrition disorders
Fatigue
|
20.0%
2/10 • Number of events 2 • 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.
|
|
Nervous system disorders
Headache
|
20.0%
2/10 • Number of events 3 • 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.
|
|
Hepatobiliary disorders
Increased alanine aminotransferase
|
20.0%
2/10 • Number of events 2 • 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.
|
|
Hepatobiliary disorders
Increased aspartate aminotransferase
|
20.0%
2/10 • Number of events 2 • 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.
|
|
Psychiatric disorders
Irritable mood
|
10.0%
1/10 • Number of events 1 • 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.
|
|
Psychiatric disorders
Jitteriness
|
10.0%
1/10 • Number of events 1 • 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.
|
|
Skin and subcutaneous tissue disorders
Mouth sores
|
10.0%
1/10 • Number of events 1 • 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.
|
|
Gastrointestinal disorders
Nausea
|
20.0%
2/10 • Number of events 3 • 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.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place