Clinical study to assess the efficacy and safety of Cannabidiol in children and young adults with rare disease-associated severe epilepsy

Prior treatment

• Subject has been part of a clinical trial involving another investigational medicinal product in the previous six months
• Current or past use of recreational or medicinal cannabis, or cannabinoid-based medications, within the three months prior to screening
• Patients with previous history of suicidal behaviour and ideation or at high suicidal risk based on clinical assessment and administration of the Columbia Suicide Severity Rating Scale (for patients 6 years of age, when appropriate otherwise, clinical judgment will be used)
• Subject taking more than four concurrent ASMs, corticotropins in the six months prior to screening, felbamate for less than one year prior to screening

Other illness

• Known hypersensitivity to CBD or any of the excipients in the study formulation
• Progressive neurological disease
• Clinically significant unstable medical conditions other than epilepsy 5. Any other significant disease or disorder which, in the opinion of the investigator, may either put the patient at risk because of participation in the study, may influence the result of the study, or affect the patient’s ability to participate in the study

Lab values

• Impaired hepatic function at screening defined as any of the following: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) greater than 3 times the upper limit of normal (ULN) and total bilirubin (TBL) greater than 2 times the ULN

Age

• Age <2 years

Other

• Inadequate supervision by parents and/or caregivers as judged by the investigator
• Patients with LGS, DS or TSC
• Female patients who are pregnant and female of childbearing potential unless willing to ensure the use of a highly effective method of birth control during the study and for three months thereafter

• Male or female aged 2-25 years as of the day of the Screening Visit;
• Subject with rare disease-associated severe epilepsy. Subject has been certified by the National Health System as affected by a rare disease listed in https://www.malattierare.gov.it
• Patient has severe epilepsy, with at least 4 motor (generalized, focal, or both) seizures per month during baseline period, despite 2 or more current or prior ASMs;
• Previous treatment with at least 2 ASMs and currently taking at least 1 other ASMs or between one and four ASMs, with a stable antiseizure treatment for the previous 4 weeks (including ketogenic diet and vagal nerve stimulation);
• Subject’s parent/caregiver has been informed of the nature of the study and informed consent has been obtained from the legally responsible parent/guardian;
• Subject’s parent/caregiver is willing and able to be compliant with diary completion, visit schedule and study drug accountability in the opinion of the investigator.
• Surgical treatment for the epilepsy has failed or cannot be pursued