Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
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|ClinicalTrials.gov Identifier: NCT02332655|
Recruitment Status : Completed
First Posted : January 7, 2015
Results First Posted : March 2, 2022
Last Update Posted : March 2, 2022
|Condition or disease||Intervention/treatment||Phase|
|Sturge-Weber Syndrome||Drug: Cannabidiol||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||5 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome|
|Study Start Date :||December 2014|
|Actual Primary Completion Date :||April 2019|
|Actual Study Completion Date :||April 2021|
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 48 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 epilepsy in patients with Sturge-Weber syndrome.
Initiation of treatment will begin with 2mg/kg/day. The dose will be increased by 3 mg/kg/day after seven days and then by 5 mg/kg/day every seven days up to a maximum dose of 25 mg/kg/day given.
The dose of concomitant antiepileptic drugs will remain unchanged during the first 12 weeks of CBD treatment (or until 8 weeks after steady state at final dose), unless symptoms of toxicity and/or significant changes in blood levels are observed.
- Number of Seizures Per Month [ Time Frame: Measured within 56 days before baseline and 56 days before week 14 ]A baseline seizure frequency was recorded for each subject in a diary for eight weeks prior to investigational drug initiation and parents/caregivers documented seizures on a daily basis throughout the trial using a seizure log. For assessing the efficacy of CBD, the investigator counted the change in frequency of seizures per month. The number of seizures within 56 days of the baseline and the number of seizures within 56 days of week 14 were calculated. Higher seizure frequency indicates worse outcome. This outcome is measured as the change in number of seizures per month between the baseline and week 14 time points.
- Percentage Change in Seizure Frequency at Most Recent Visit on CBD Compared With Baseline [ Time Frame: Measured at Baseline and most recent visit within 1 year ]The percentage change, between the seizure frequency per month reported at baseline compared to seizure frequency per month at the subject's most recent visit, on CBD was calculated. Higher positive percentage change in seizure frequency per month would indicate better outcome. Positive values indicate a decrease in seizure frequency.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02332655
|United States, Maryland|
|Kennedy Krieger Institute|
|Baltimore, Maryland, United States, 21205|
|Principal Investigator:||Anne M Comi, MD||Hugo W. Moser Research Institute at Kennedy Krieger, Inc.|