Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial registered on ANZCTR
Registration number
ACTRN12624000948594
Ethics application status
Approved
Date submitted
1/07/2024
Date registered
5/08/2024
Date last updated
1/09/2024
Date data sharing statement initially provided
5/08/2024
Type of registration
Prospectively registered
Titles & IDs
Public title
Efficacy And Withdrawal Symptoms in Transition Between Cannabidivarin (CBDV) and Cannabidiol (CBD) in Children with Rett Syndrome and Refractory Epilepsy
Query!
Scientific title
Efficacy And Withdrawal Symptoms in Transition Between CBDV to CBD in Children with Rett Syndrome and Refractory Epilepsy
Query!
Secondary ID [1]
312430
0
Nil known
Query!
Universal Trial Number (UTN)
U1111-1310-1734
Query!
Trial acronym
Query!
Linked study record
The previous phase I trial will have the same participants for the current study. The publication of the phase I is: Hurley EN, Ellaway CJ, Johnson AM, Truong L, Gordon R, Galettis P, Martin JH, Lawson JA. Efficacy and safety of cannabidivarin treatment of epilepsy in girls with Rett syndrome: A phase 1 clinical trial. Epilepsia. 2022 Jul;63(7):1736-1747. doi: 10.1111/epi.17247. Epub 2022 Apr 20
Query!
Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome
334246
0
Query!
Refractory Epilepsy
334321
0
Query!
Condition category
Condition code
Neurological
330911
330911
0
0
Query!
Epilepsy
Query!
Neurological
330980
330980
0
0
Query!
Other neurological disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
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.
Query!
Intervention code [1]
328936
0
Treatment: Drugs
Query!
Comparator / control treatment
(1) baseline on CBDV (4 week screening period). The screening period will have patents record prospective seizures in the seizure diary.
Query!
Control group
Active
Query!
Outcomes
Primary outcome [1]
338674
0
Seizure Frequency
Query!
Assessment method [1]
338674
0
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
Query!
Timepoint [1]
338674
0
13 weeks, 1 year (primary), 3 years post transition commencement.
Query!
Secondary outcome [1]
436976
0
global impression of change
Query!
Assessment method [1]
436976
0
global impression of change score
Query!
Timepoint [1]
436976
0
1 year post-transition commencement
Query!
Secondary outcome [2]
436977
0
CBD withdrawal symptoms
Query!
Assessment method [2]
436977
0
Cannabis Withdrawal Scale
Query!
Timepoint [2]
436977
0
13 weeks post-transition commencement
Query!
Secondary outcome [3]
436978
0
Sleep
Query!
Assessment method [3]
436978
0
Pittsburgh Sleep Quality Index
Query!
Timepoint [3]
436978
0
13 weeks post-transition commencement
Query!
Eligibility
Key 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.
Query!
Minimum age
12
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Females
Query!
Can healthy volunteers participate?
No
Query!
Key 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.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Non-randomised trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 2
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Date of first participant enrolment
Anticipated
12/08/2024
Query!
Actual
21/08/2024
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
Query!
Sample size
Target
3
Query!
Accrual to date
1
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
NSW
Query!
Recruitment hospital [1]
26745
0
Sydney Children's Hospital - Randwick
Query!
Recruitment postcode(s) [1]
42792
0
2031 - Randwick
Query!
Funding & Sponsors
Funding source category [1]
316847
0
Commercial sector/Industry
Query!
Name [1]
316847
0
Jazz Pharmaceuticals
Query!
Address [1]
316847
0
Query!
Country [1]
316847
0
Australia
Query!
Primary sponsor type
Hospital
Query!
Name
Sydney Children's Hospital Network
Query!
Address
Query!
Country
Australia
Query!
Secondary sponsor category [1]
319081
0
None
Query!
Name [1]
319081
0
Query!
Address [1]
319081
0
Query!
Country [1]
319081
0
Query!
Ethics approval
Ethics application status
Approved
Query!
Ethics committee name [1]
315610
0
Sydney Children's Hospitals Network Human Research Ethics Committee
Query!
Ethics committee address [1]
315610
0
http://www.schn.health.nsw.gov.au/health-professionals/our-research/ethics-research-governance
Query!
Ethics committee country [1]
315610
0
Australia
Query!
Date submitted for ethics approval [1]
315610
0
Query!
Approval date [1]
315610
0
06/06/2023
Query!
Ethics approval number [1]
315610
0
2023/ETH00474
Query!
Summary
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.
Query!
Trial website
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
135242
0
Dr Alexandra Johnson
Query!
Address
135242
0
Sydney Children's Hospital, High St, Randwick NSW 2031
Query!
Country
135242
0
Australia
Query!
Phone
135242
0
+612 9382 1547
Query!
Fax
135242
0
Query!
Email
135242
0
[email protected]
Query!
Contact person for public queries
Name
135243
0
Kaitlyn Griffin
Query!
Address
135243
0
Sydney Children's Hospital, High St, Randwick NSW 2031
Query!
Country
135243
0
Australia
Query!
Phone
135243
0
+612 9382 5376
Query!
Fax
135243
0
Query!
Email
135243
0
[email protected]
Query!
Contact person for scientific queries
Name
135244
0
Kaitlyn Griffin
Query!
Address
135244
0
Sydney Children's Hospital, High St, Randwick NSW 2031
Query!
Country
135244
0
Australia
Query!
Phone
135244
0
+612 9382 5376
Query!
Fax
135244
0
Query!
Email
135244
0
[email protected]
Query!
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
Query!
No/undecided IPD sharing reason/comment
Pooled deidentified results will be released post analysis upon publication (if applicable).
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
Documents added manually
No documents have been uploaded by study researchers.
Documents added automatically
No additional documents have been identified.
Download to PDF