Clinical trial NCT02910297: The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy
Official title: The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Dravet Syndrome
Lennox Gastaut Syndrome
Keywords: cannabidiol
Dravet Syndrome
Lennox Gastaut Syndrome
Lead sponsor: Gillette Children's Specialty Healthcare
Sponsor class: OTHER
Collaborators: name: University of Minnesota
First posted: 2016-09-22
Enrollment: 0
Sex: ALL
Minimum age (years): 2
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: CASE_CROSSOVER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects who have registered or plan to register with the state of Minnesota for taking medical cannabis and have severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome)

Brief summary: The purpose of this study is to determine if cannabidiol (CBD) obtained via the state of Minnesota reduces seizures in patients with severe intractable epilepsy (Dravet Syndrome or Lennox Gastaut Syndrome), and to measure blood levels of CBD to help determine CBD concentration-response characteristics.

Detailed description: Patients with medically refractory epilepsy who are registering to take CBD in the State of Minnesota will be offered the opportunity to participate in this study. If consent to participation is obtained, the patient (or more likely, the patient's guardian) will be asked to begin a seizure diary for at least a 7-day period prior (ideally, longer) to receiving the CBD, and to continue tracking seizures throughout the study. Upon receipt of CBD from the Minnesota dispensary, subjects will take their CBD as directed and will return to the clinic for a repeat visit (which is part of standard care) occurring 2-8 weeks after continual use of CBD. During these clinic visits, patients undergo blood draws to monitor their other prescribed AED concentrations as part of standard of care. In addition to the standard blood draw, the research participant will be asked to provide a blood sample of 5 ml to measure CBD concentrations. As most patients are instructed to take CBD two hours prior to their standard of care AEDs, clinical experience indicates that most patients are taking CBD quite early in the morning (around 6 AM). Thus, the time of sample collection will be recorded as hours post-dose, and will likely be nearer peak serum concentrations. If a study participant has an indwelling intravenous access port, they will be asked to provide an additional sample of 5 ml at least one hour after the initial sample. During the repeat visit, subjects will be asked to continue documenting seizure activity in their seizure diary while they continue their CBD treatment. Any changes to AEDs administered concurrently with CBD will also be recorded.

Subjects who are already on a stable regimen of CBD will also be eligible to participate. At a clinic visit, they will be asked to provide a blood sample of 5 ml, the time of each sample will be recorded as hours post-dose of CBD. They will also be asked to begin a seizure diary and continue tracking seizure activity. If a patient terminates their CBD treatment, they will be asked to continue tracking seizure activity after their last CBD dose until their follow-up clinic visit, to determine change in seizure activity while on and off CBD.

Eligibility criteria: Inclusion Criteria:

* Diagnosed with Dravet Syndrome or Lennox-Gastaut Syndrome
* Patients who are planning to obtain medical cannabidiol
* Patients who are already taking medical cannabidiol and are planning to stop taking it.

Exclusion Criteria:

* Patients without a diagnosis of Dravet syndrome or Lennox-Gastaut syndrome

Primary outcomes:
measure: Reduction in seizures
timeFrame: a 7-day seizure diary completed at least twice within 3 months
Secondary outcomes:
measure: Cannabidiol concentration
timeFrame: 1 time at a clinic visit within three months. If a patient has a port, they will be asked to provide an additional blood sample of 5 ml at least one hour after the first sample during the same clinic visit.
Other outcomes:
Locations:
Study officials:
affiliation: Gillette Children's Specialty Healthcare
name: Samuel A Roiko, Ph.D.
role: PRINCIPAL_INVESTIGATOR
References: