None listed
Conditions
Brief summary
All the patients with Rett Syndrome, previously on CBDV as part of the phase I trial, will be offered transition to Epidyolex, with monitoring for change in seizure frequency and severity, sleep behaviours and emergence of withdrawal symptoms. The study hypothesis is that patients with refractory epilepsy and Rett syndrome will respond to CBD (similar seizure frequency, seizure severity) when transitioned from CBDV.
Interventions
Efficacy And Withdrawal Symptoms in Transition Between Cannabidivarin (CBDV) and Cannabidiol (CBD) in Children with Rett Syndrome and Refractory Epilepsy
Treatment regime (oral dosing):
Day 0-7: CBDV 7.5mg/kg/day and CBD 2.5mg/kg/day
Day 8-14: CBDV 5mg/kg/day and CBD 5mg/kg/day
Day 15-21: CBDV 2.5mg/kg/day and CBD 7.5mg/kg/day
Day 22-28: CBDV 0mg/kg/day and CBD 10mg/kg/day
onward: CBD 10-20mg/kg/day depending on response from patient.
Adherence: weight of returned CBD/CBDV bottle.
Sponsors
Sydney Children's Hospital Network
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
All
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Involvement in previous phase I trial of CBDV in Rett Syndrome. 2. Rett syndrome with known MECP2 mutation. 3. Refractory epilepsy (having failed an adequate trial of at least two standard anti-seizure medications). 4. Patient and caregiver willing and able to comply with all trial requirements. 5. All medications and interventions stable for four weeks prior to screening and patient / caregiver willing to maintain stable regimen throughout trial.
Exclusion criteria
1. Another significant neurological diagnosis (history of traumatic brain injury, metabolic disease, or infection). 2. Significant non-neurological diagnosis (e.g., severe cardiac or respiratory disease). 3. Pre-existing abnormalities of full blood count, electrolytes, coagulation, hepatic function or enzymes considered clinically significant as judged by the investigator (e.g., WCC < 4, platelets < 60 000, ANC < 1, ALT or AST > 2 times upper limit normal). 4. Clinically significant ECG abnormality (e.g., QTc > 460 msec, PR > 0.2 sec). 5. Patient currently using or has used other cannabinoid products other than CBDV and unwilling to abstain for duration of the trial. 6. Female subjects who are pregnant will be excluded. A negative serum pregnancy test is required at screening. If female subjects become pregnant during the study, they must inform the investigator, and consult an obstetrician. 7. Known allergy to or any component of either CBDV, CBD or any cannabinoid. 8. Patient has any other significant disease or disorder which in the opinion of the investigator, may put the patient at risk or influence the result of the trial or the patient’s ability to participate in the trial. 9. Any abnormalities identified following a physical examination of the patient that, in the opinion of the investigator, would jeopardize the safety of the patient if they took part in the trial. 10. Patient is taking more than four other concurrent anti-epileptic drugs. 11. Patient has taken felbamate in year prior to screening.
Outcome results
None listed