Cannabidiol on beyond-Seizure outcomes in children and adults with Developmental and Epileptic Encephalopathy
A pilot study to investigate the effect of Cannabidiol on quality of life and beyond-seizure outcomes in children and adults with Developmental and Epileptic Encephalopathy
Hunter New England Local Health District
20 participants
Jul 31, 2024
Interventional
Conditions
Summary
The BEYOND Study is a multi-centre, open-label pilot study that utilises a participant-centric clinical trial protocol for epilepsy. It aims to assess the effectiveness, safety, and tolerability of Epidyolex as an adjunctive therapy for beyond seizure outcomes in individuals aged 2 to 65 years with DEE. The study will enrol 20 participants from metropolitan and regional areas in New South Wales, including rural and remote locations. The acceptability of this model will help guide health economists and medical affairs teams in collecting cost-effective data that can be used to optimise Pharmaceutical Benefits Advisory Committee (PBAC) submissions with regulators.
Eligibility
Inclusion Criteria12
- 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.
- Male or female (non-pregnant, non-lactating female) aged 2 years € to 65 years old.
- 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.
- 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)
- The Participant/parent/caregiver is able and willing to complete study visits, complete the questionnaires, record concomitant medications, and take study drugs as instructed.
- 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 Criteria13
- Seizures secondary to an infection, neoplasia, demyelinating disease, degenerative neurological disease, or central nervous system disease deemed progressive, metabolic illness, or progressive degenerative disease.
- History or presence of any significant medical or surgical condition other than a DEE
- History of jaundice which lasts longer than 1 week will require exclusion of hepatic disease before entering the study.
- Subjects who are known to have a terminal illness.
- 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.
- Subjects who are planning to begin to follow a ketogenic or other specialized dietary therapy during the study.
- 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.
- Concurrent enrolment in any other type of medical research judged by the Study Doctor not to be scientifically or medically compatible with this study.
- 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.
- Subjects who are currently taking adrenocorticotropic hormone.
- Subjects who did not tolerate Cannabidiol when taken previously or were allergic to sesame seed/oil.
- 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.
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Interventions
This is an open-labelled trial to assess the health outcomes of Cannabidiol (CBD) administered as Epidiolex. Epidiolex is a purified cannabidiol oral solution that will be administered orally or via gastrostomy and nasogastric feeding tubes. All participants will undergo a screening process. Eligible participants will begin 48 weeks of treatment with CBD. Each treatment block includes 2 weeks of titration, 46 weeks on the maintenance dose and 10 days of drug tapering. Treatment group A (Epidiolex) Parents/caregivers or patients will administer the study drug orally/via gastrostomy/via a nasogastric tube in two divided doses (12 hours +/- 2 hours) daily. Dosage will be titrated over 2 weeks until the maximum (20 mg/kg/day) or best tolerated dose is reached. The maximum or best-tolerated dose will be maintained for 46 weeks. Dosing schedule: Day 1: 1.25 mg/kg/day via two divided doses Day 2: 2.5mg/kg/day via two divided doses Day 3: 5mg/kg/day via two divided doses Day 4: 5mg/kg/day via two divided doses Day 5: 7.5 mg/kg/day via two divided doses Day 6: 7.5 mg/kg/day via two divided doses Day 7: 10mg/kg/day via two divided doses Day 8: 10mg/kg/day via two divided doses Day 9: 15mg/kg/day via two divided doses Day 10: 15mg/kg/day via two divided doses Day 11: 20 mg/kg/day via two divided doses Day 12: 20 mg/kg/day via two divided doses Day 13: 20 mg/kg/day via two divided doses Day 14: 20 mg/kg/day via two divided doses Compliance with study medication will be reviewed at each virtual or in-clinic appointment. Pharmacy staff will calculate the expected number of bottles (based on participants weight and expected titration schedule) required between visit time points and dispense to the participants accordingly via Direct To Patient service. Home nurses will be required to account all used and/or any unused bottles of medication and report to the study site pharmacy where clinical trial pharmacists will complete accountability and adherence calculations. This information will be forwarded to the PI for noting. A new prescription for the study medication will be provided by the PI to the clinical trial pharmacists who will dispense a new supply of study medication to see participants through until their next appointment. Taper schedule: Day 1: 20 mg/kg/day via two divided doses Day 2: 17.5 mg/kg/day via two divided doses Day 3: 15 mg/kg/day via two divided doses Day 4:12.5 mg/kg/day via two divided doses Day 5: 10 mg/kg/day via two divided doses Day 6: 7.5 mg/kg/day via two divided doses Day 7: 5 mg/kg/day via two divided doses Day 8: 2.5 mg/kg/day via two divided doses Day 9: 1.35 mg/kg/day via two divided doses Day 10: 0 mg/kg/day
Locations(1)
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ACTRN12624000888561