Trial · NCT04447846

Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome

NCT04447846, Phase 2, Completed, Anne Comi, MD; results first posted 2023-05-25

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome. Trials, record NCT04447846. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04447846

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Novel Cognitive Treatment Targets for Epidiolex in Sturge- Weber Syndrome
NCT number: NCT04447846
Status: Completed
Phases: Phase 2
Enrollment: 11 participants, actual
Lead sponsor: Anne Comi, MD
Conditions: Sturge-Weber Syndrome
Interventions: Cannabidiol
First posted: 2020-06-25
Last updated: 2023-05-25
Results first posted: 2023-05-25
Limitations and caveats: 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.

Source ClinicalTrials.gov

Registered outcomes19

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	List Sorting Working Memory Test	Baseline, Follow-up (6 months)	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.
Secondary	Picture Vocabulary Test	Baseline, Follow-up (6 months)	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.
Secondary	Seizure Frequency	Baseline, Follow-up (6 months)	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.
Secondary	Migraine Severity	Baseline, Follow-up (6 months)	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.
Secondary	Modified House Classification Scores	Baseline, Follow-up (6 months)	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.
Secondary	Erhardt Developmental Prehension Assessment Scores	Baseline, Follow-up (6 months)	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.
Secondary	Pediatric Evaluation of Disability Inventory Computer Adapted Test	Baseline, Follow-up (6 months)	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.
Secondary	ABILHAND Questionnaire	Baseline, Follow-up (6 months)	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.
Secondary	Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)	Baseline, Follow-up (6 months)	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.
Secondary	Pediatric Neurological Quality of Life (Neuro-QoL)	Baseline, Follow-up (6 months)	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.

1 to 10 of 19Source ClinicalTrials.gov

See all 19 registered outcomes

Outcomes19

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	List Sorting Working Memory Test	Baseline, Follow-up (6 months)	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.	T-score	MEAN
SECONDARY	Picture Vocabulary Test	Baseline, Follow-up (6 months)	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.	T-score	MEAN
SECONDARY	Seizure Frequency	Baseline, Follow-up (6 months)	Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.	score on a scale	MEAN
SECONDARY	Migraine Severity	Baseline, Follow-up (6 months)	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.	score on a scale	MEAN
SECONDARY	Modified House Classification Scores	Baseline, Follow-up (6 months)	Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.	score on a scale	MEAN
SECONDARY	Erhardt Developmental Prehension Assessment Scores	Baseline, Follow-up (6 months)	Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.	Months	MEAN
SECONDARY	Pediatric Evaluation of Disability Inventory Computer Adapted Test	Baseline, Follow-up (6 months)	Subject 9 was withdrawn prior to the 6 month follow-up visit due to a persistent side effect and was not included in analysis.	score on a scale	MEAN
SECONDARY	ABILHAND Questionnaire	Baseline, Follow-up (6 months)	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.	score on a scale	MEAN
SECONDARY	Wechsler Intelligence Scale for Children, Fifth Edition (WISC-V) or Wechsler Adult Intelligence Scale (WAIS-IV)	Baseline, Follow-up (6 months)	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.	score on a scale	MEAN
SECONDARY	Pediatric Neurological Quality of Life (Neuro-QoL)	Baseline, Follow-up (6 months)	Subjects below the age of 18 completed the pediatric version of this questionnaire and were included in analysis.	T-score	MEAN

1 to 10 of 19Source ClinicalTrials.gov

See all 19 outcomes

Results by group110

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
List Sorting Working Memory Test	Cannabidiol/ Epidiolex	Baseline List Sorting Working Memory Fully Adjusted Score	37.14	Standard Deviation 10.92	T-score
List Sorting Working Memory Test	Cannabidiol/ Epidiolex	Follow-Up List Sorting Working Memory Fully Adjusted Score	39.14	Standard Deviation 13.78	T-score
Picture Vocabulary Test	Cannabidiol/ Epidiolex	Baseline Mean of the Fully Adjusted Picture Vocabulary Score	42.88	Standard Deviation 8.86	T-score
Picture Vocabulary Test	Cannabidiol/ Epidiolex	Follow-up Mean of the Fully Adjusted Picture Vocabulary Score	42.38	Standard Deviation 9.43	T-score
Seizure Frequency	Cannabidiol/ Epidiolex	Baseline Seizure Score from Sturge-Weber Neuroscore	1.11	Standard Deviation 0.33	score on a scale
Seizure Frequency	Cannabidiol/ Epidiolex	Follow-up Seizure Score from Sturge-Weber Neuroscore	1.00	Standard Deviation 0.00	score on a scale
Migraine Severity	Cannabidiol/ Epidiolex	Baseline Mean Migraine-Quality of Life Score	96.83	Standard Deviation 7.62	score on a scale
Migraine Severity	Cannabidiol/ Epidiolex	Follow-up Mean Migraine-Quality of Life Score	97.50	Standard Deviation 4.69	score on a scale
Modified House Classification Scores	Cannabidiol/ Epidiolex	Baseline Mean Highest Category in Which All Items were Successfully Completed	5.11	Standard Deviation 0.93	score on a scale
Modified House Classification Scores	Cannabidiol/ Epidiolex	Follow-up Mean Highest Category in Which All Items were Successfully Completed	5.44	Standard Deviation 1.33	score on a scale

1 to 10 of 110Source ClinicalTrials.gov

See all 110 results by group

Participant flow3

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Cannabidiol/ Epidiolex	10
Overall Study	COMPLETED	Cannabidiol/ Epidiolex	9
Overall Study	NOT COMPLETED	Cannabidiol/ Epidiolex	1

Source ClinicalTrials.gov

Adverse events12

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Darkening of port-wine birthmark	Skin and subcutaneous tissue disorders	Cannabidiol/ Epidiolex	1	10	1
other	Decreased appetite	Metabolism and nutrition disorders	Cannabidiol/ Epidiolex	1	10	3
other	Diarrhea	Gastrointestinal disorders	Cannabidiol/ Epidiolex	2	10	2
other	Dizziness	Ear and labyrinth disorders	Cannabidiol/ Epidiolex	2	10	3
other	Fatigue	Metabolism and nutrition disorders	Cannabidiol/ Epidiolex	2	10	2
other	Headache	Nervous system disorders	Cannabidiol/ Epidiolex	2	10	3
other	Increased alanine aminotransferase	Hepatobiliary disorders	Cannabidiol/ Epidiolex	2	10	2
other	Increased aspartate aminotransferase	Hepatobiliary disorders	Cannabidiol/ Epidiolex	2	10	2
other	Irritable mood	Psychiatric disorders	Cannabidiol/ Epidiolex	1	10	1
other	Jitteriness	Psychiatric disorders	Cannabidiol/ Epidiolex	1	10	1

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse events

Adverse event totals3

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Cannabidiol/ Epidiolex	0	10
Total, other adverse events	Cannabidiol/ Epidiolex	7	10
Total, serious adverse events	Cannabidiol/ Epidiolex	0	10

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests4

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

Test	Named as	Source
Size Order Memory Test	Primary outcome	ConductCognition
Digit Span Test	Secondary outcome	ConductCognition
Symbol Scan Task	Secondary outcome	ConductCognition
Vocabulary Test	Secondary outcome	ConductCognition

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

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