Clinical trial NCT02461706: Treatment of Drug Resistant Epilepsy
Official title: Physician Expanded Access to Investigational New Drug Cannabidiol for the Treatment of Drug Resistant Epilepsy
Study type: EXPANDED_ACCESS
Overall status: NO_LONGER_AVAILABLE
Conditions: Epilepsy
Keywords: Drug resistant epilepsy
Lead sponsor: University of Florida
Sponsor class: OTHER
Collaborators: name: James and Esther King Biomedical Research Program
First posted: 2015-06-03
Sex: ALL
Minimum age (years): 2
Maximum age (years): 16
Standard ages: CHILD

Brief summary: The main goal of this study is to provide treatment with cannabidiol (CBD) to children with drug resistant epilepsy through a Physician Expanded Access Investigation New Drug protocol. As the controlled therapeutic use of CBD in children is novel, the primary objective of this study is to evaluate the safety and efficacy of CBD when administered as adjunctive therapy in children that have exhausted the majority of FDA approved antiepileptic drug treatment options.

Detailed description: Fifty, (50) children will be provided CBD as part of the Principal Investigator's Physician Expanded Access IND. CBD will be administered as an adjunct to all current antiepileptic drugs. After the screening visit, a 4 week baseline period will be followed and seizures will be recorded in the seizure diary given to the patient at this time. The patients will be clinically evaluated at baseline, once a month for three months and once every three months thereafter. For the first week after study initiation, patients will be called at day 3 to review any changes in condition and again at day 7 to review any changes in condition and to determine whether an increase in dosing is required. Subsequently, patients will be called each week during the titration period to review any changes in condition and determine whether an increase in dosing is required. Maximal dose titration should be achieved in most patients within 5 weeks. In order to ensure safe use at higher doses, patients who receive more than 600 mg of daily CBD will be evaluated at least monthly until they achieve steady state at their final fixed maintenance dose. The investigator will be available by telephone or email throughout the initial four months of the study in between the visits.

Eligibility criteria: Inclusion Criteria:

* history of a trial of at least four drugs, including one trial of a combination of two concomitant drugs, without successful seizure control.
* Vagal nerve stimulation: VNS must be on stable settings for a minimum of 1 month,
* RNS deep brain stimulation, or
* the ketogenic diet can be considered equivalent to a drug trial and must be on a stable ratio for a minimum of 3 months.
* anti-epileptic drugs at stable doses for a minimum of 4 weeks prior to enrollment.
* 4 clinically countable seizures per month: Seizure history to include a documented history of generalized seizures (drop attacks, atonic, tonic-clonic and/or myoclonic), focal seizures without loss of consciousness with a motor component, focal seizures with loss of consciousness, or focal seizures with secondary generalization,

Exclusion Criteria:

* Use of CBD based product within the previous 30 days.
* Initiation of felbamate within 6 months.
* Allergy to CBD or any cannabinoid.
* Unable to comply with study visits/requirements.
* Diagnosis of Dravet Syndrome or Lennox-Gastaut syndrome will be excluded completely from this trial.
* Cannabidiol is contraindicated in pregnancy. Female subjects who are pregnant will be excluded from the study. If a female subject is able to become pregnant, she will be given a serum pregnancy test before entry into the study. Female subjects will be informed not become pregnant while taking cannabidiol. Female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if they become pregnant during the study.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Flordia CRC
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital, Miami Children's Health System
state: Florida
Study officials:
affiliation: University of Florida
name: Steven Winesett, MD
role: PRINCIPAL_INVESTIGATOR
References: