Cannabidiol on beyond-Seizure outcomes in children and adults with Developmental and Epileptic Encephalopathy

Phase 1

• Conditions: Epilepsy; Neurological - Epilepsy

• Registration Number: ACTRN12624000888561
• Lead Sponsor: Hunter New England Local Health District

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-07-22
• Last Update Posted: 29 July 2024

Recruitment & Eligibility

• Status: ot yet recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

1.Has a documented clinical diagnosis of Dravet syndrome, Lennox-Gastaut syndrome as confirmed by the Pharmaceutical Benefits Scheme (PBS), or other developmental and epileptic encephalopathy ß.
2.Male or female (non-pregnant, non-lactating female) aged 2 years € to 65 years old.
3.Have countable motor seizures *, defined as:
-an average of greater than or equal to (>=) 2 per month during the past 3 months, based on the Study Doctor's assessment
-and a monthly average of >=1 per month during the Baseline Period, based on the seizure diary record, documented by caregiver report or Study Doctor medical notes.
4.Subjects can be taking 1 and no more than 3 concomitant antiseizure medications (ASMs) a. All doses must be stable for at least 4 weeks before screening and expected to be maintained throughout the study (until the end of the treatment period)
5.The Participant/parent/caregiver is able and willing to complete study visits, complete the questionnaires, record concomitant medications, and take study drugs as instructed.
6.For participants with G-tube/Percutaneous endoscopic gastrostomy (PEG) tube, G-tubes/PEG tubes should have been placed and been functioning for at least 3 months before screening.

* drop seizures, tonic-clonic, tonic, bilateral clonic, atonic, myoclonic-atonic, myoclonic-tonic-clonic, focal seizures with bilateral hyperkinetic motor features

a Epilepsy surgery is allowed and will not be counted as a concomitant ASM. A ketogenic diet is allowed and will not be counted as a concomitant ASM. The subject must be on a stable dietary regimen that produces ketosis for at least 6 weeks before screening and is expected to be maintained throughout the study.

ß CDKL5 Deficiency Disorder, SCN2A, SYNGAP1, West Syndrome, STXBP1, Ring20 Chromosome disorder, Rett Syndrome and DEPDC5.

€ The minimum age is due to the recommended age limit for using the portable electroencephalogram (EEG) device. Subjects between the ages of 2-6 years are eligible if their parent/guardian can provide an EEG completed in the last 6 months at the Baseline and the end of the trial.

Exclusion Criteria

1.Seizures secondary to an infection, neoplasia, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease.
2.History or presence of any significant medical or surgical condition other than a DEE
3.History of jaundice which lasts longer than 1 week will require exclusion of hepatic disease before entering the study.
4.Subjects who are known to have a terminal illness.
5.Any clinically significant laboratory abnormalities or clinically significant abnormalities on pre-study physical examination, vital signs, or ECG that in the judgment of the Study Doctor indicate a medical problem that would preclude study participation.
6.Subjects who are planning to begin to follow a ketogenic or other specialized dietary therapy during the study.
7.Exposure to any other investigational drug or device within 5 half-lives or 30 days before screening (Visit 1), whichever is longer or plans to participate in another drug or device trial at any time during the study.
8.Concurrent enrolment in any other type of medical research judged by the Study Doctor not to be scientifically or medically compatible with this study.
9.Subjects using felbamate who have presented with clinically significant abnormalities and/or hepatic dysfunction during felbamate treatment, and subjects who have taken felbamate for less than 6 months before screening.
10.Subjects who are currently taking adrenocorticotropic hormone.
11.Subjects who did not tolerate Cannabidiol when taken previously or were allergic to sesame seed/oil.
12.Subjects who have a Vagal Nerve Stimulator (VNS) device. µ

µ Subjects with a VNS can be eligible if an EEG can be provided to the study doctor at the beginning and the end of the study.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
•To assess the effect of Epidyolex ® on Quality of Life in participants with Developmental and Epileptic Encephalopathy (DEE) and their caregivers.[•A change in overall EQ-5D-5L score at 48 weeks compared with baseline. Baseline and 48 weeks post-baseline]