A Phase 2 interventional study of Cannabidiol in Sturge-Weber Syndrome, sponsored by Anne Comi, MD. Completed at 1 site in United States. Open to participants aged 3 Years to 50 Years. Per ClinicalTrials.gov, last updated 2023-05-25.
Sponsored by Anne Comi, MD · Phase 2, Interventional, and Treatment
Among 1,418 studies in Cognitive Dysfunction with a site in United States — 328 open to participants now, 432 under way, 13 of them phase 3. Cognitive Dysfunction · United States.
Among the 1,418 studies in Cognitive Dysfunction with a site in United States, this one is the 1,023rd largest by enrollment of the 1,143 interventional ones, is one of 89 that started in 2019, is one of 198 in phase 2, and was the 706th registered.
3,872 studies on the registry are indexed under Cognitive Dysfunction; 1,119 are open to participants now.
This study's enrollment of 11 is below the median of 64 across 2,794 interventional studies indexed under Cognitive Dysfunction.
Browse Cognitive Dysfunction studies →328 of the 1,418 studies in Cognitive Dysfunction with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 750 Cognitive Dysfunction studies recruiting now, 362 (48%) set no upper age limit and 143 (19%) stop at 75 or younger; 46 take children under 18; 259 take healthy volunteers.
This study: 3 Years to 50 Years · all sexes · no healthy volunteers.
The full table on the hub →1,418 studies; the count open now beside each. This study's row is marked.
First in 1993; this study’s year is marked. 89 started in 2019.
Median enrollment 58 across the 1,143 interventional studies, 150 across the 270 observational. Of the 885 that completed or were terminated, 377 (43%) have results posted.
209 of 1,418 have an industry lead sponsor. The 10 most frequent:
Anne Comi, MD is the lead sponsor of 3 studies on the registry; none are open to participants now.
Anne Comi, MD has 2 studies filed under Cognitive Dysfunction — fewer than ten, so there is no comparison to draw.
180 of the studies in Cognitive Dysfunction with a site in United States that are under way reach primary completion within a year, 4 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT03860857Phase 3University of California, IrvineBiomarker Exploration in Aging, Cognition and Neurodegeneration | 2026-12-22 |
| NCT03887455Phase 3Eisai Inc.A Study to Confirm Safety and Efficacy of Lecanemab in Participants With Early Alzheimer's Disease | 2026-12-31 |
| NCT06191965Phase 3Mclean HospitalMitoQ for Early-phase Schizophrenia-spectrum Disorder and Mitochondrial Dysfunction | 2027-01-31 |
| NCT06950190Phase 3Ellis MedicineEvaluating Cognitive Changes on Patients in Chemotherapy (ECCPC) | 2027-02-20 |
| NCT03560960Boston UniversityMulti-Center Development of a Novel Diagnostic Test for Alzheimer's Disease | 2026-11 |
Among the 1,418 studies in Cognitive Dysfunction with a site in United States, from their registry updates:
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.
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:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: 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.
Also known as: Epidiolex, CBD
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.
Time frame: Baseline, Follow-up (6 months)
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, Follow-up (6 months)
| Milestone | Cannabidiol/ Epidiolex |
|---|---|
| Started | 10 |
| Completed | 9 |
| Not completed | 1 |
| Withdrew: Withdrew due to moderate side effects (fatigue) | 1 |
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.
| T-score | Cannabidiol/ Epidiolex |
|---|---|
| Baseline List Sorting Working Memory Fully Adjusted Score | 37.14 ± 10.92 |
| Follow-Up List Sorting Working Memory Fully Adjusted Score | 39.14 ± 13.78 |
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.
| T-score | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean of the Fully Adjusted Picture Vocabulary Score | 42.88 ± 8.86 |
| Follow-up Mean of the Fully Adjusted Picture Vocabulary Score | 42.38 ± 9.43 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Seizure Score from Sturge-Weber Neuroscore | 1.11 ± 0.33 |
| Follow-up Seizure Score from Sturge-Weber Neuroscore | 1.00 ± 0.00 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Migraine-Quality of Life Score | 96.83 ± 7.62 |
| Follow-up Mean Migraine-Quality of Life Score | 97.50 ± 4.69 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Highest Category in Which All Items were Successfully Completed | 5.11 ± 0.93 |
| Follow-up Mean Highest Category in Which All Items were Successfully Completed | 5.44 ± 1.33 |
| Baseline Mean Total Number of Items Successfully Completed | 26.67 ± 4.12 |
| Follow-up Mean Total Number of Items Successfully Completed | 27.44 ± 4.75 |
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.
| Months | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Dominant Hand Total Score | 54.22 ± 2.64 |
| Follow-up Mean Dominant Hand Total Score | 53.56 ± 4.04 |
| Baseline Mean Non-Dominant Hand Total Score | 51.22 ± 9.48 |
| Follow-up Mean Non-Dominant Hand Total Score | 53.78 ± 4.15 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Score for Daily Activities | 58.67 ± 7.76 |
| Follow-up Mean Score for Daily Activities | 60.00 ± 6.50 |
| Baseline Mean Score for Mobility | 67.11 ± 4.76 |
| Follow-Up Mean Score for Mobility | 68.56 ± 4.07 |
| Baseline Mean Score for Responsibility | 52.44 ± 11.67 |
| Follow-Up Mean Score for Responsibility | 53.89 ± 11.10 |
| Baseline Mean Score for Social Cognitive | 66.89 ± 5.35 |
| Follow-up Mean Score for Social Cognitive | 67.22 ± 4.63 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean of the ABILHAND | 1.81 ± 1.98 |
| Follow-up Mean of the ABILHAND | 2.88 ± 1.39 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Digit Span Score | 5.71 ± 2.81 |
| Follow-up Mean Digit Span Score | 6.43 ± 2.07 |
| Baseline Mean Symbol Search Score | 6.00 ± 2.71 |
| Follow-up Mean Symbol Search Score | 5.57 ± 2.07 |
| Baseline Mean Coding Score | 5.57 ± 2.88 |
| Follow-up Mean Coding Score | 4.71 ± 2.22 |
| Baseline Mean Processing Speed Score | 77.14 ± 13.93 |
| Follow-up Mean Processing Speed Score | 73.43 ± 11.19 |
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.
| T-score | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Pediatric Score for Anger | 53.02 ± 3.09 |
| Follow-up Mean Pediatric Score for Anger | 49.67 ± 7.00 |
| Baseline Mean Pediatric Score for Anxiety | 45.20 ± 5.79 |
| Follow-up Mean Pediatric Score for Anxiety | 41.07 ± 5.74 |
| Baseline Mean Pediatric Score for Pain | 40.20 ± 4.16 |
| Follow-up Mean Pediatric Score for Pain | 43.50 ± 5.89 |
| Baseline Mean Pediatric Score for Social Relations | 43.28 ± 15.10 |
| Follow-up Mean Pediatric Score for Social Relations | 54.33 ± 6.96 |
| Baseline Mean Pediatric Score for Stigma | 42.45 ± 6.42 |
| Follow-up Mean Pediatric Score for Stigma | 44.52 ± 4.88 |
| Baseline Mean Pediatric Score for Depression | 44.07 ± 6.29 |
| Follow-up Mean Pediatric Score for Depression | 44.62 ± 8.58 |
| Baseline Mean Pediatric Score for Cognitive Function | 49.27 ± 3.36 |
| Follow-up Mean Pediatric Score for Cognitive Function | 51.33 ± 6.44 |
| Baseline Mean Pediatric Score for Fatigue | 46.38 ± 9.44 |
| Follow-up Mean Pediatric Score for Fatigue | 45.97 ± 8.14 |
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.
| T-score | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean for Behavioral Regulation Index | 55.11 ± 11.66 |
| Follow-Up Mean for Behavioral Regulation Index | 52.44 ± 13.60 |
| Baseline Mean for Emotional Regulation Index | 60.22 ± 10.24 |
| Follow-up Mean for Emotional Regulation Index | 51.22 ± 12.29 |
| Baseline Mean for Cognitive Regulation Index | 54.11 ± 7.06 |
| Follow-up Mean for Cognitive Regulation Index | 54.22 ± 10.59 |
| Baseline Mean for Global Executive Composite | 56.78 ± 8.17 |
| Follow-up Mean for Global Executive Composite | 53.33 ± 11.57 |
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.
| T-score | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Total Score | 54.22 ± 7.71 |
| Follow-up Mean Total Score | 49.22 ± 7.09 |
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.
| T-score | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean External Problems Composite | 51.22 ± 6.10 |
| Follow-up Mean External Problems Composite | 50.11 ± 6.31 |
| Baseline Mean Internal Problems Composite | 52.22 ± 5.81 |
| Follow-Up Mean Internal Problems Composite | 46.22 ± 7.17 |
| Baseline Mean Behavioral Symptoms Index | 54.22 ± 4.21 |
| Follow-up Mean Behavioral Symptoms Index | 50.11 ± 8.19 |
| Baseline Mean Adaptive Skills Composite | 45.00 ± 8.29 |
| Follow-Up Mean Adaptive Skills Composite | 48.44 ± 7.63 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean of Total Score | 14.86 ± 14.49 |
| Follow-up Mean of Total Score | 11.57 ± 11.16 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Total Score | 61.17 ± 18.14 |
| Follow-up Mean Total Score | 71.39 ± 10.75 |
Safety of Epidiolex was measured by the number of adverse events and serious adverse events that result from study drug.
| Events | Cannabidiol/ Epidiolex |
|---|---|
| Number of Adverse Events that Resulted from the Study Drug | 24 |
| Number of Serious Adverse Events that Resulted from the Study Drug | 0 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Seizure Score | 1.11 ± 0.33 |
| Follow-Up Mean Seizure Score | 1.00 ± 0.00 |
| Baseline Mean Hemiparesis Score | 1.44 ± 1.13 |
| Follow-Up Mean Hemiparesis Score | 1.00 ± 0.87 |
| Baseline Mean Visual Field Cut Score | 0.11 ± 0.33 |
| Follow-Up Mean Visual Field Cut Score | 0.00 ± 0.00 |
| Baseline Mean Cognitive Function Score | 2.33 ± 0.50 |
| Follow-up Mean Cognitive Function Score | 1.44 ± 0.53 |
| Baseline Mean Composite Score | 5.00 ± 1.32 |
| Follow-up Mean Composite Score | 3.44 ± 0.88 |
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.
| score on a scale | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Total Score | 8.56 ± 3.09 |
| Follow-up Mean Total Score | 8.11 ± 3.18 |
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.
| T-score | Cannabidiol/ Epidiolex |
|---|---|
| Baseline Mean Adult Score for Anxiety | 46.77 ± 13.33 |
| Follow-Up Mean Adult Score for Anxiety | 46.87 ± 6.45 |
| Baseline Mean Adult Score for Depression | 42.70 ± 10.05 |
| Follow-Up Mean Adult Score for Depression | 44.23 ± 9.53 |
| Baseline Mean Adult Score for Fatigue | 45.23 ± 9.53 |
| Follow-Up Mean Adult Score for Fatigue | 42.80 ± 4.60 |
| Baseline Mean Adult Score for Upper Extremity Function | 48.97 ± 8.37 |
| Follow-Up Mean Adult Score for Upper Extremity Function | 53.80 ± 0.00 |
| Baseline Mean Adult Score for Lower Extremity Function | 58.60 ± 0.00 |
| Follow-Up Mean Adult Score for Lower Extremity Function | 58.60 ± 0.00 |
| Baseline Mean Adult Score for Cognitive Function | 47.03 ± 15.52 |
| Follow-Up Mean Adult Score for Cognitive Function | 46.73 ± 5.01 |
| Baseline Mean Adult Score for Emotional and Behavioral Dyscontrol | 51.10 ± 14.97 |
| Follow-Up Mean Adult Score for Emotional and Behavioral Dyscontrol | 41.37 ± 9.98 |
| Baseline Mean Adult Score for Well-being | 54.30 ± 4.91 |
| Follow-Up Mean Adult Score for Well-being | 55.70 ± 6.59 |
| Baseline Mean Adult Score for Sleep Disturbances | 47.03 ± 7.49 |
| Follow-Up Mean Adult Score for Sleep Disturbances | 43.93 ± 4.90 |
| Baseline Mean Adult Score for Participation in Social Activities | 53.00 ± 7.41 |
| Follow-Up Mean Adult Score for Participation in Social Activities | 51.03 ± 8.01 |
| Baseline Mean Adult Score for Satisfaction in Social Activities | 47.70 ± 3.28 |
| Follow-Up Mean Adult Score for Satisfaction in Social Activities | 49.23 ± 2.54 |
| Baseline Mean Adult Score for Stigma | 53.90 ± 8.80 |
| Follow-Up Mean Adult Score for Stigma | 56.47 ± 6.25 |
Collected over 6 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cannabidiol/ Epidiolex | 0/10 (0%) | 0/10 (0%) | 7/10 (70%) |
| Event | Cannabidiol/ Epidiolex |
|---|---|
| DiarrheaGastrointestinal disorders | 2/10 |
| DizzinessEar and labyrinth disorders | 2/10 |
| FatigueMetabolism and nutrition disorders | 2/10 |
| HeadacheNervous system disorders | 2/10 |
| Increased alanine aminotransferaseHepatobiliary disorders | 2/10 |
| Increased aspartate aminotransferaseHepatobiliary disorders | 2/10 |
| NauseaGastrointestinal disorders | 2/10 |
| Darkening of port-wine birthmarkSkin and subcutaneous tissue disorders | 1/10 |
| Decreased appetiteMetabolism and nutrition disorders | 1/10 |
| Irritable moodPsychiatric disorders | 1/10 |
| Age, Continuous(years) | Cannabidiol |
|---|---|
| Mean | 13.83 ± 9.67 |
| Sex: Female, Male(Participants) | Cannabidiol |
|---|---|
| Female | 6 |
| Male | 4 |
| Ethnicity (NIH/OMB)(Participants) | Cannabidiol |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 7 |
| Unknown or Not Reported | 1 |
| Race (NIH/OMB)(Participants) | Cannabidiol |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 1 |
| Black or African American | 0 |
| White | 6 |
| More than one race | 2 |
| Unknown or Not Reported | 1 |
| Region of Enrollment(participants) | Cannabidiol |
|---|---|
| United States | 10 |
| Brain Involvement(Participants) | Cannabidiol |
|---|---|
| Right-Sided Brain Involvement | 6 |
| Left-Sided Brain Involvement | 2 |
| Bilateral Brain Involvement | 2 |
| Skin Involvement(Participants) | Cannabidiol |
|---|---|
| Right-Sided Skin Involvement | 4 |
| Left-Sided Skin Involvement | 2 |
| Bilateral Skin Involvement | 4 |
| Eye Involvement(Participants) | Cannabidiol |
|---|---|
| Right-Sided Eye Involvement | 4 |
| Left-Sided Eye Involvement | 2 |
| Bilateral Eye Involvement | 1 |
| No Eye Involvement | 3 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Oct 2019 to primary completion in Aug 2021, 22 months.
Results were first posted in May 2023, 1 year 9 months after primary completion.
Of 145 non-industry phase 2 interventional studies under Cognitive Dysfunction, half reached primary completion within 53 months of starting (the median), counting those still running; a quarter within 29 and three quarters within 64. This study's 22 months is below that.
Counting every sponsor, half of 186 studies reached it within 44 months; a quarter within 22 and three quarters within 62.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in May 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Cannabidiol