Safety and Tolerability of Cannabidiol in Subjects With Drug Resistant Epilepsy
Safety and Tolerability of Cannabidiol in Subjects With Drug Resistant Epilepsy
This is an observational, open-label, flexible dose study that will prospectively and
 longitudinally assess the effect of Cannabidiol (CBD) therapy in patients with drug-resistant
 epilepsies through a Physician Expanded Access Investigational New Drug protocol.
This study will test if Cannabidiol (CBD) therapy is safe in humans and reduces the number
 and/or severity of seizures in patients with drug resistant epilepsy when taken in addition
 to current anti-epileptic drugs (AEDs).

 To be eligible, subjects must:

 -  have drug-resistant epilepsy

 -  be between 1 and 60 years of age

 Subjects may participate for up to 2 years and may continue to receive the drug as available,
 until the compassionate use is terminated or the drug becomes approved by the FDA.

 The study includes:

 -  Baseline, Screening and 2 phone calls

 -  12 week drug titration (6 visits) and 5 phone calls

 -  Approximately 1 year and 9 month Treatment period (7 visits)

 Subjects will come in for visits approximately every two weeks for the first three months and
 then quarterly for the remainder of the study. Subjects' antiepileptic drug levels will be
 tested to monitor for drug interactions. Lab assessments (CBC, CMP, urinalysis) will be
 performed to monitor for changes in bone marrow, liver, and kidney function to ensure safe
 use.

 Cannabidiol will be in the form of 100 mg/ml oral solution. Subjects will be given CBD to
 take in addition to their current anti-epileptic drug (AED) regimen.
Inclusion Criteria:

 1. Ages 1-60 years of age.

 2. Patient must have at least 4 clinically countable seizures per month. They must also
 have prior concomitant video-EEG with evidence documenting a diagnosis of epilepsy.
 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.

 3. Drug resistant epilepsy defined as a trial of at least four drugs, including one trial
 of a combination of two concomitant drugs, without successful seizure control. Vagal
 nerve stimulation, RNS deep brain stimulation, or the ketogenic diet can be considered
 equivalent to a drug trial and documented evidence of drug and other therapeutic
 failures.

 4. Between 1-4 baseline anti-epileptic drugs at time of enrollment. Vagus nerve
 stimulator (VNS), ketogenic diet and modified Atkins diet do not count toward this
 limit and are not contraindicated for inclusion.

 5. Subject and family sign assent (if capable)/consent/research authorization and are
 able to meet the study expectations for appointments for the duration of the study

 6. VNS, if in use must be on stable settings for a minimum of 1 month.

 7. If on ketogenic diet, must be on stable ratio for a minimum of 3 months.

 8. Patients or their caregivers must be able to consistently maintain a seizure diary for
 at least 2 months prior to enrollment and during the course of the study period.

 9. Must be Nebraska state resident.

 Exclusion Criteria:

 1. Renal, hepatic, pancreatic, or hematologic dysfunction as evidenced by: values above
 upper limits of normal for BUN/creatinine, or values twice the upper limit of normal
 for serum transaminases (ALT/SGPT, AST/SGOT), values twice the upper limit of normal
 for serum lipase and amylase, platelets <80,000 /mcL, WBC<3.0 103 /mcL

 2. Less than 4 countable (non-countable seizures includes absence and myoclonic) seizures
 per month

 3. Use of cannabis-related product within last 30 days

 4. Active substance abuse/addiction.

 5. CBD is contraindicated in pregnancy and breastfeeding. 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 urine pregnancy test before entry into the study. Female
 subjects will be informed not to become pregnant while taking cannabidiol, and must
 agree to an acceptable method of barrier contraception use during the study which
 should include abstinence or a double barrier method for the duration of treatment.

 Female subjects must tell the investigator and consult an obstetrician or
 maternal-fetal specialist if they become pregnant during the study. If pregnancy
 occurs, CBD will be stopped in the most clinically appropriate manner.

 6. Allergy to CBD or any cannabinoid.

 7. Unable to provide consent (and no LAR available)

 8. Unable to comply with study visits/requirements.

 9. Use of alcohol