Epidiolex in Typical Absence Seizures

• Status: Enrolling by invitation
• Trial end date: 2021-10-05
• Target enrollment: 0
• Participant gender: All

Summary

This is a Pilot study, open-label study consisting of a screening period of up to 4 weeks, a 4-week dose-titration treatment period to dose of up to 20 mg/kg/day BID of CBD (Epidiolex), and a 30 day safety follow-up period following the last dose of study medication.

• Phase: Phase 4
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: University of South Florida
• Collaborator: Greenwich Biosciences
• Treatments: Cannabidiol; Epidiolex

Criteria

10.1 Existing clinical patients diagnosed with Genetic (Idiopathic) Generalized Epilepsy will be reviewed and asked if they are willing to participate.

10.2 Signed informed consent form (ICF) indicating that the subject has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side effects, risks, and discomforts.

Men or non-pregnant, non-breastfeeding women 16 to 55 years of age who are able to read and understand the written and spoken local language.

Clinical diagnosis of IGE (including, but not limited to, CAE, JAE, juvenile myoclonic epilepsy, or Jeavons syndrome) with absence seizures consistent with the International League against Epilepsy Revised Classification of Seizures (2017).

Absence seizures, by history on average once per hour, persisting despite standard of care (SOC) treatment, defined as treatment with at least 2 AEDs appropriate for the patient's epilepsy syndrome. SOC failure, per investigator discretion, will be defined as insufficient clinical response or intolerable side effects, which precludes use of the appropriate AED.

Observation of at least 3 instances of generalized discharges of approximately 2.5 - 4 Hz lasting >2 seconds via 24-hr ambulatory EEG

On stable doses of one or more antiepileptic medication(s) for at least 30 days. If a subject is not on medication, adequate documentation justifying lack of therapy may be acceptable for the subject after Greenwich Biosciences review. Ketogenic, modified Atkins diet (MAD), or low glycemic diet with stable carbohydrate ratio for at least 30 days before screening is an acceptable antiepileptic therapy. Vagal nerve stimulation at stable settings (for at least 30 days before screening), without use of the magnet, is also acceptable.

Subjects with reproductive capability, including all males and WOCBP, must agree to practice continuous abstinence or adequate contraception methods (appropriate double barrier method or oral, patch, implant, or injectable contraception) from as soon as feasible during screening period until at least 30 days after the last dose (i.e., intermittent abstinence based on "rhythm," temperature monitoring, or other means of timing is not acceptable). WOCBP include any woman who has experienced menarche and who has not undergone successful surgical sterilization (hysterectomy, bilateral tubal ligation, and/or bilateral oophorectomy) or is not postmenopausal. Postmenopausal is defined as amenorrhea for ≥12 consecutive months without another cause, and a documented serum follicle stimulating hormone (FSH) level ≥35 mIU/mL.

Able and willing to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.

10.3 Subjects will be excluded from the study if any of the following conditions apply.

History of surgical intervention for treatment of epilepsy.

Additional seizure (clinical and electrographic) types, including, but not limited to, epileptic spasms, generalized tonic seizures, atonic seizures, or focal seizures. Subjects with GTCS or myoclonic seizures are eligible for the study.

Inadequately treated psychotic or mood disorder (e.g., schizophrenia, major depression, bipolar disorder).

Presence of severe intellectual disability, severe autism spectrum disorder, or severe developmental disorder such that the subject cannot sign the ICF or cannot cooperate with the study procedures.

Presence of positive urine drug screen for drugs of abuse, except if this is explained by use of an allowed prescription medicine.

Significant hepatic (AST/ALT or bilirubin ≥2 × ULN) or renal disease (creatinine clearance

≤39 mL/min).

A current C-SSRS score of 4 or 5 at screening or history of suicide attempt within the past year. Study team members have been trained on the administration of this assessment.

Any other condition and/or situation that causes the investigator to deem a subject unsuitable for the study.

Treatment with an investigational agent within 30 days prior to the first dose of CBD (Epidiolex) or planning to receive an investigational agent during the study.

Concurrent legal use of CBD obtained from state dispensaries.

10.5 Students, Employees, those that are socially and/or economically disadvantaged and Wards of the state will not be included in this research population.