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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05184478




Registration number
NCT05184478
Ethics application status
Date submitted
2/12/2021
Date registered
11/01/2022

Titles & IDs
Public title
Is Medicinal Cannabis an Effective Treatment for Tourette Syndrome in Adolescents? a Pilot Study
Scientific title
A Pilot Randomized Placebo-controlled Crossover Trial of Medicinal Cannabis (MC) in Adolescents with Tourette Syndrome (TS)
Secondary ID [1] 0 0
RCH HREC 69238
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Tourette Syndrome in Adolescence 0 0
Condition category
Condition code
Mental Health 0 0 0 0
Other mental health disorders
Neurological 0 0 0 0
Other neurological disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Medicinal cannabis (MC): THC 10mg/mL : CBD 15mg/mL, manufactured by Cann Group Ltd.
Treatment: Drugs - Placebo

Experimental: Group A (MC then placebo) - Group A will receive medicinal cannabis during Treatment Period 1 (70 days), followed by a 7 day dose reduction and 21 day wash-out period, then will receive placebo during Treatment Period 2 (70 days).

Experimental: Group B (placebo then MC) - Group B will receive placebo during Treatment Period 1 (70 days), followed by a 7 day dose reduction and 21 day wash-out period, then will receive medicinal cannabis during Treatment Period 2 (70 days).


Treatment: Drugs: Medicinal cannabis (MC): THC 10mg/mL : CBD 15mg/mL, manufactured by Cann Group Ltd.
Each 1mL contains 10mg tetrahydrocannabinol (THC),15 mg cannabidiol (CBD), and 0.004mL peppermint oil in medium chain triglyceride (MCT) oil.

All participants will start at 0.1 mL per day, and will gradually increase in 0.1mL increments until day 21 when a dose of 0.5mL (participants weighing \<50kg) or 1mL (participants weighing =50kg) is reached. At day 29, an assessment of treatment response will be conducted. Participants who meet criteria for a treatment response will remain on the same dose for the remainder of the treatment period. Participants who do not meet criteria for a treatment response will gradually increase the dose in 0.1mL increments until day 49 when a daily dose of 1mL (participants weighing \<50kg) or 2mL (participants =50kg) is reached.

Treatment: Drugs: Placebo
The placebo contains MCT oil and peppermint flavoring solution, which is indistinguishable from the active medication in appearance, smell and taste.

The dose will be matched for volume to the medicinal cannabis, and will follow the same dosing schedule as the medicinal cannabis treatment phase.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rate of study participant recruitment, calculated as the time required to reach a sample size of 10.
Timepoint [1] 0 0
From the date of pre-screening the first participant until the tenth participant is randomized, up to 2 years.
Primary outcome [2] 0 0
Participant withdrawal rate, calculated as the number of participants who withdraw from the trial as a proportion of the total number of participants randomized.
Timepoint [2] 0 0
Day 1 to day 176 (end of treatment period 2)
Primary outcome [3] 0 0
Study medication tolerability, as indicated by the proportion of participants who tolerate the protocol dosing schedule.
Timepoint [3] 0 0
Day 1 to day 176 (end of treatment period 2)
Primary outcome [4] 0 0
Participant adherence to the study medication dosing schedule, calculated as the proportion of participants who demonstrate acceptable medication compliance.
Timepoint [4] 0 0
Day 78 (end of treatment period 1) and day 176 (end of treatment period 2)
Primary outcome [5] 0 0
Study visit attendance, calculated as the proportion of visits completed across the study sample.
Timepoint [5] 0 0
Screening to day 169 (final study visit)
Primary outcome [6] 0 0
Blood test completion, calculated as the proportion of blood tests completed across the study sample.
Timepoint [6] 0 0
Screening to day 169 (final study visit)
Primary outcome [7] 0 0
Parent questionnaire completion, calculated as the proportion of parent-report questionnaires completed across the study sample.
Timepoint [7] 0 0
Screening to day 169 (final study visit)
Primary outcome [8] 0 0
Self-report questionnaire completion, calculated as the proportion of adolescent self-report questionnaires completed across the study sample.
Timepoint [8] 0 0
Screening to day 169 (final study visit)
Primary outcome [9] 0 0
Study design acceptability will be evaluated through a parent-completed study specific evaluation questionnaire completed at the end of the study.
Timepoint [9] 0 0
Day 197
Secondary outcome [1] 0 0
The frequency of adverse events as reported on the modified version of the Liverpool Adverse Event Profile (LAEP) at day 71 and day 169 will be summarized across the medicinal cannabis and placebo treatment phases.
Timepoint [1] 0 0
Day 71 and 176
Secondary outcome [2] 0 0
The frequency of adverse events as reported throughout the study will be summarized across the medicinal cannabis and placebo treatment phases.
Timepoint [2] 0 0
Day 1 to day 197

Eligibility
Key inclusion criteria
* Males and females aged 12 - 18 years of age;
* DSM-5 (Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition) diagnosis of TS as assessed by the study clinician;
* TS severity defined as a score of 20 or higher on the Total Tic Severity section of the Yale Global Tic Severity Scale;
* No changes in either medication or other interventions in the 4 weeks prior to randomization, and intention to remain on 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: clobazam, mTOR (mammalian target of rapamycin) inhibitors (e.g sirolimus, tacrolimus), anti-cancer agents, citalopram >20mg/day, escitalopram >10mg/day;
* Abnormal liver function tests defined as ALT (alanine transaminase) > twice ULN (upper limit of normal);
* 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
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/05/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/01/2024
Sample size
Target
Accrual to date
Final
10
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital / Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville

Funding & Sponsors
Primary sponsor type
Other
Name
Murdoch Childrens Research Institute
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
The de-identified data set collected for this analysis of the MC in TS Pilot Study will be available six months after publication of the primary outcome. The study protocol and analysis plan will also be available. The data must be obtained from the Murdoch Children's Research Institute. Prior to releasing any data the following are required: a data access agreement must be signed between relevant parties, the 'MC in TS Pilot Study' Study Management Group must see and approve the data analysis plan describing how the data will be analysed, there must be an agreement around appropriate acknowledgment and any additional costs involved must be covered. Should the Study Management Group be unavailable, this role is delegated to the Murdoch Children's Research Institute. Data will only be shared with a recognized research organisation which has approved the proposed analysis plan.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP)
When will data be available (start and end dates)?
6 months after publication of primary outcome
Available to whom?
1) Data access agreement; 2) approval by Trial Steering Committee; 3) recognized research institutions.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05184478