Cannabidiol in Children and Young Adults With Rare Disease-associated Severe Epilepsy

NCT05803434 | Unknown Phase 2

• 1 other identifier: CBD_RE
• Study Type: interventional
• Target: 30
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a pilot, open-label, phase II study. The main objective of the study is to demonstrate that Cannabidiol (CBD), used in addition to current anti-seizure medications (ASMs) reduces the number and/or severity of motor (generalized, focal, or both) seizures in children and young adults with rare disease-associated severe epilepsy. Secondary objectives include assessment of safety and tolerability, changes in behaviour, cognition and sleep, pharmacokinetic interaction with concurrent ASMs.

Conditions

Epilepsy; Rare Diseases

Keywords

drug resistant epilepsy; rare epilepsy; cannabidiol

Outcome Measures

Primary Outcomes (2)

• Change in number of generalized and/or focal motor-onset seizure frequency

  percentage change per 28 days from the 4-week baseline period in generalized and/or focal motor-onset seizure frequency during the 24-week treatment period

  24 weeks

• Change in severity of generalized and/or focal motor-onset seizure frequency

  a score will be established for each patient, based on review and comparison of all baseline-EEG/7-weeks control-EEG and baseline-EEG/15-weeks control-EEG, with values ranging from 0 (= worsened EEG), to a maximum of 2 (= improved); 1 will be assigned if the EEG trace is unmodified

  24 weeks

Secondary Outcomes (10)

• Incidence of adverse events

  24 weeks

• Body weight

  24 weeks

• Maximum Plasma Concentraion [Cmax] of concurrent ASMs

  24 weeks

• Number of subjects considered treatment responders

  24 weeks

• Number of subjects who are free of motor (generalized, focal, or both) seizures

  24 weeks

Study Arms (1)

Cannabidiol treatment

EXPERIMENTAL

Drug: Cannabidiol oral solution

Interventions

Cannabidiol oral solution DRUG

Cannabidiol will be administered orally twice daily into equally divided doses. The starting dose is 2.5 mg/kg twice daily. The dose can be gradually increased to 5 mg/kg twice daily, which is the recommended maintenance dose, up to a maximum dose of 10 mg/kg twice daily, according to tolerability and clinical response. Following titration, subjects will continue treatment over a 20-week maintenance period. The total treatment duration from the beginning of the titration period till the end of the maintenance period will be 24 weeks.

Cannabidiol treatment

Eligibility Criteria

• Age: 2 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Male or female;
• Children (age 2-18 years) and young adults (18-25 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;
• 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

You may not qualify if:

• Age \<2 years;
• Known hypersensitivity to CBD or any of the excipients in the study formulation;
• Progressive neurological disease;
• Clinically significant unstable medical conditions other than epilepsy that may place patient's safety at risk;
• 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;
• 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;
• Subject taking more than four concurrent ASMs;
• Subject has taken corticotropins in the six months prior to screening;
• Subjects taking felbamate, and they have been taking it for less than one year prior to screening;
• Inadequate supervision by parents and/or caregivers as judged by the investigator;
• 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 and is unwilling to abstain for the duration for the study;
• Female patients who are pregnant;
• Female patients of childbearing potential or male patient whose partner is of childbearing potential, unless willing to ensure that they or their partner use a highly effective method of birth control during the study and for three months thereafter.

Sponsors & Collaborators

• Meyer Children's Hospital IRCCS: lead

MeSH Terms

Conditions

Epilepsy; Rare Diseases; Drug Resistant Epilepsy

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Central Study Contacts

Renzo Guerrini, MD, FRCP, FAES

CONTACT

00390555662573; renzo.guerrini@meyer.it

Simona Balestrini, MD, PhD

CONTACT

00390555662718; simona.balestrini@meyer.it

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Head of Child Neurology Department, Principal Investigator, Clinical Professor

Study Record Dates

• First Submitted: February 28, 2023
• First Posted: April 7, 2023
• Study Start: June 1, 2023
• Primary Completion: March 1, 2025
• Study Completion: March 1, 2025
• Last Updated: April 18, 2023

Record last verified: 2023-04