Efficacy And Withdrawal Symptoms in Transition Between Cannabidivarin (CBDV) and Cannabidiol (CBD) in Children with Rett Syndrome and Refractory Epilepsy
- Conditions
- Rett SyndromeRefractory EpilepsyNeurological - EpilepsyNeurological - Other neurological disorders
- Registration Number
- ACTRN12624000948594
- Lead Sponsor
- Sydney Children's Hospital Network
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Female
- Target Recruitment
- 3
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.
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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Seizure Frequency[Treatment responders (to CBD) defined as showing < 25% increase in seizure frequency on CBD at 1 year from baseline compared to seizure frequency (on CBDV) via a seizure diary 13 weeks, 1 year (primary), 3 years post transition commencement. ]
- Secondary Outcome Measures
Name Time Method global impression of change [global impression of change score 1 year post-transition commencement];CBD withdrawal symptoms[Cannabis Withdrawal Scale 13 weeks post-transition commencement];Sleep [Pittsburgh Sleep Quality Index 13 weeks post-transition commencement]