Cannabidiol Expanded Access Study in Medically Refractory Sturge-Weber Syndrome
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT02332655|
Recruitment Status : Active, not recruiting
First Posted : January 7, 2015
Last Update Posted : January 8, 2019
|Condition or disease||Intervention/treatment||Phase|
|Sturge-Weber Syndrome||Drug: Cannabidiol||Phase 1 Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||10 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|
|Estimated Primary Completion Date :||June 2019|
|Estimated Study Completion Date :||June 2019|
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.
- Change in seizure frequency [ Time Frame: 1 year ]Seizure Frequency between visits and per week will be calculated
- Change in average seizure duration by seizure type [ Time Frame: 1 year ]Average seizure duration in seconds and minutes for each seizure type experienced by subject.
- Change in the number of episodes of status epilepticus, defined as convulsive seizure lasting longer than 10 minutes [ Time Frame: 1 year ]Any episodes of status epilepticus are noted and changes in the number experienced by subject over the 1 year time frame are calculated.
- Change in the number of uses of rescue medication [ Time Frame: 1 year ]Each time a rescue medication is used for a seizure between visits the data is noted and changes in frequency used over the 1 year time period will be calculated.
- Change in the number of ER visits/ hospitalizations [ Time Frame: 1 year ]Number of ER visits or hospitalizations will be recorded and changes in frequency will be calculated.
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.|