Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12622000031763
Ethics application status
Approved
Date submitted
12/11/2021
Date registered
14/01/2022
Date last updated
20/02/2024
Date data sharing statement initially provided
14/01/2022
Date results provided
20/02/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
Phase I Pilot study of Medicinal Cannabis (MC) in adolescents with Tourette Syndrome (TS).
Scientific title
A Phase 1 Pilot open-label trial of Medicinal Cannabis (MC) in adolescents with Tourette Syndrome (TS)
Secondary ID [1] 305780 0
Nil
Universal Trial Number (UTN)
Trial acronym
Linked study record
Not Applicable

Health condition
Health condition(s) or problem(s) studied:
Tourette Syndrome 324284 0
Condition category
Condition code
Neurological 321778 321778 0 0
Other neurological disorders
Mental Health 321986 321986 0 0
Other mental health disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is an open-label pilot study of 10 participants (adolescents aged 12 to 18 years with TS) who will be treated with Medicinal Cannabis (CBD 15mg/ml, THC 10mg/Ml). Eligible participants will receive THC:CBD 10:15 oil in the Treatment period.
Participants weighing <50kg will receive a maximum THC dose of 10mg per day, and those weighing greater than or equal to 50 kg will receive a maximum THC dose of 20mg per day.
Both schedules commence with 1mg/day, followed by an initial up-titration period of 21 days.
For the lower weight schedule, the dose is increased by 1mg every 4-5 days until they reach 5mg/day. For the higher weight schedule, the dose is increased by 1mg every 2-3 days until they reach 10mg/day. Once participants have been on this dose for 7 days (i.e. Day 29), they will undergo a dose assessment using the Parent Tic Questionnaire, to determine whether they have responded to this dose.
Participants will return all study medication (including opened, unopened and empty bottles) at the end of treatment visit for each phase for compliance checking. The remaining volume of each bottle of study medication will be measured and recorded by the study coordinator and cross-checked with the prescribed volume in order to assess participant compliance.
Liver function tests will be done at screening and then repeated at the end of treatment period (Day 85).
Urine test will be done at screening for illicit drugs and medicinal cannabis. For women of child bearing potential, a pregnancy test will also be conducted at screening and on Day 85.
Intervention code [1] 322178 0
Treatment: Drugs
Comparator / control treatment
This is a open label single arm study and therefore Comparator/ control treatment is not applicable.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 329534 0
This is a composite primary outcome assessing the feasibility and acceptability of the study protocol. The outcomes that are metrics for assessing the feasibility and acceptability will be rate of recruitment that will be determined by number of patients enrolled during the recruitment period as per medical records,
Proportion of participants who withdraw from the protocol for any reasons such as study medication tolerability , safety or compliance.
Completion of key protocol elements including blood test completion.
Timepoint [1] 329534 0
Dose assessment visit at Day 29
End of treatment at day 85
side effects are monitored daily until the end of study.
Secondary outcome [1] 402906 0
Adverse Events (AE) reported on the LAEP will be considered significant if a 2-point increase is reported from baseline to end of maintenance. The frequency of AE meeting this criteria will be presented for the treatment CBD. All Serious Adverse Events will be reported. The number and proportion of participants with at least 1 AE will also be reported.
Timepoint [1] 402906 0
Compared with baseline at Day 85, D91 and D119.

Eligibility
Key inclusion criteria
Males and females aged 12 – 18 years of age;
DSM-5 diagnosis of TS as assessed by the study clinician.
TS severity defined as a score of 20 out of 50 or higher on the Total Tic Severity section of the YGTSS;
No changes in either medication or other interventions in the 4 weeks prior to enrolment, and intention to remain on the same dose for the duration of the study;
Participant and family have the ability to comply with the protocol requirements, in the opinion of the investigator;
Agrees not to drive for the duration of the study.
Minimum age
12 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Non-English speaking parents;
Participant history of psychosis, schizophrenia, bipolar disorder, or major depressive disorder; or a family history of psychosis;
Taking anti-epileptic medications which interact with medicinal cannabis (e.g. clobazam, topiramate, zonisamide);
Abnormal liver function tests defined as ALT > twice ULN;
Current use of illicit drugs or medicinal cannabis, or use in the 4 weeks prior to screening
Pregnant or intending to become pregnant during the study, or breastfeeding.
History of clinically significant suicidal thoughts in the prior 12 months.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Not applicable as this is an open label study
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Not applicable
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis
As this is a pilot study, all data will be analysed using descriptive statistics.With regard to feasibility outcomes, data will be presented as frequencies and percentages of the events. (Frequency of discontinuations, percentage completion of protocol elements). The recruitment rate will be presented as the percentage of participants that are consented out of the eligible participants determined to meet criteria at screening, including those who decide not to proceed. The reasons for not participating will be summarised as the number stating each reason. The acceptability of enrolment, tolerability of the study medication, acceptability of the study visits and procedures will be presented as mean scores with ranges / standard deviations. Withdrawals, treatment discontinuations and protocol violations will be summarised.

This study is not powered to detect treatment effects. Therefore, questionnaire data will be reported using descriptive statistics. We will use paired-t test to evaluate the trend of effectiveness for outcomes that are continuous variables. The effect size estimates will also be used to calculate required sample size to design the full-scale clinical trial as a follow-up study.

Adverse Events (AE) reported on the LAEP will be considered significant if a 2-point increase is reported from baseline to end of maintenance. The frequency of AE meeting this criteria will be presented for the treatment CBD and placebo groups respectively. All Serious Adverse Events will be reported. The number and proportion of participants with at least 1 AE will also be reported.

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
1/02/2022
Actual
9/09/2022
Date of last participant enrolment
Anticipated
15/12/2023
Actual
15/06/2023
Date of last data collection
Anticipated
26/04/2024
Actual
11/10/2023
Sample size
Target
10
Accrual to date
Final
10
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 21096 0
Liverpool Hospital - Liverpool
Recruitment postcode(s) [1] 35949 0
2170 - Liverpool

Funding & Sponsors
Funding source category [1] 310135 0
University
Name [1] 310135 0
University of New South Wales
Country [1] 310135 0
Australia
Primary sponsor type
Hospital
Name
South Western Sydney Local Health District
Address
Administration Building, Eastern Campus, Liverpool Hospital Locked Bag 7279, Liverpool BC NSW 1871
Country
Australia
Secondary sponsor category [1] 311213 0
None
Name [1] 311213 0
Address [1] 311213 0
Country [1] 311213 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 309825 0
Sydney Children's Hospital Network Research Ethics Committee
Ethics committee address [1] 309825 0
Ethics committee country [1] 309825 0
Australia
Date submitted for ethics approval [1] 309825 0
23/08/2021
Approval date [1] 309825 0
28/09/2021
Ethics approval number [1] 309825 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 115514 0
Prof Valsamma Eapen
Address 115514 0
ICAMHS, L1 MHC, Liverpool Hospital, Elizabeth Street, NSW 2170
Sydney, Australia
Country 115514 0
Australia
Phone 115514 0
+61 2 9616 4346
Fax 115514 0
Email 115514 0
[email protected]
Contact person for public queries
Name 115515 0
Valsamma Eapen
Address 115515 0
ICAMHS, L1 MHC, Liverpool Hospital, Elizabeth Street, NSW 2170
Sydney, Australia
Country 115515 0
Australia
Phone 115515 0
+61 2 9616 4346
Fax 115515 0
Email 115515 0
[email protected]
Contact person for scientific queries
Name 115516 0
Valsamma Eapen
Address 115516 0
ICAMHS, L1 MHC, Liverpool Hospital, Elizabeth Street, NSW 2170
Sydney, Australia
Country 115516 0
Australia
Phone 115516 0
+61 2 9616 4346
Fax 115516 0
Email 115516 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
IPD will not be available to others. Data analysis will be done within the investigators team.


What supporting documents are/will be available?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
14486Study protocol  [email protected]



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.