Clinical trial NCT05184478: Is Medicinal Cannabis an Effective Treatment for Tourette Syndrome in Adolescents? a Pilot Study
Official title: A Pilot Randomized Placebo-controlled Crossover Trial of Medicinal Cannabis (MC) in Adolescents with Tourette Syndrome (TS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Tourette Syndrome in Adolescence
Lead sponsor: Murdoch Childrens Research Institute
Sponsor class: OTHER
First posted: 2022-01-11
Enrollment: 10
Sex: ALL
Minimum age (years): 12
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Intervention model description: Eligible participants will be randomized 1:1 to initially receive either THC:CBD 10:15 oil or placebo in Treatment Period 1, before crossing over and receiving the other study drug (THC:CBD 10:15 oil or placebo) in Treatment Period 2.
Primary purpose: TREATMENT

Brief summary: This is a single site, pilot double-blind, randomized, placebo-controlled, cross-over study of 10 participants comparing medicinal cannabis (THC:CBD 10:15 oil) with placebo in reducing tics in adolescents aged 12 - 18 years with severe Tourette Syndrome (TS).

The primary objective of this pilot study is to evaluate all elements of the study design (recruitment strategy, study duration, study procedures, study medication tolerance and outcome measures) to assess if they are acceptable and feasible for the conduct of a full-scale randomized controlled trial of THC:CBD 10:15 oil to reduce tic severity in adolescents with TS.

The secondary objective of this study is to collect preliminary data on the safety of oral THC:CBD 10:15 oil in adolescents aged 12 to 18 years with TS.

As an exploratory aim data from clinician- and parent-rated measures will be compared across the phases to explore for a signal of efficacy on primary (tic reduction) and secondary (premonitory urges, obsessive compulsive behaviors, Attention Deficit Hyperactivity Disorder \[ADHD\] symptoms) outcome measures.


Eligibility criteria: 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.

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.

Primary outcomes:
measure: Rate of study participant recruitment, calculated as the time required to reach a sample size of 10.
timeFrame: From the date of pre-screening the first participant until the tenth participant is randomized, up to 2 years.
measure: Participant withdrawal rate, calculated as the number of participants who withdraw from the trial as a proportion of the total number of participants randomized.
timeFrame: Day 1 to day 176 (end of treatment period 2)
measure: Study medication tolerability, as indicated by the proportion of participants who tolerate the protocol dosing schedule.
timeFrame: Day 1 to day 176 (end of treatment period 2)
measure: Participant adherence to the study medication dosing schedule, calculated as the proportion of participants who demonstrate acceptable medication compliance.
timeFrame: Day 78 (end of treatment period 1) and day 176 (end of treatment period 2)
measure: Study visit attendance, calculated as the proportion of visits completed across the study sample.
timeFrame: Screening to day 169 (final study visit)
measure: Blood test completion, calculated as the proportion of blood tests completed across the study sample.
timeFrame: Screening to day 169 (final study visit)
measure: Parent questionnaire completion, calculated as the proportion of parent-report questionnaires completed across the study sample.
timeFrame: Screening to day 169 (final study visit)
measure: Self-report questionnaire completion, calculated as the proportion of adolescent self-report questionnaires completed across the study sample.
timeFrame: Screening to day 169 (final study visit)
measure: Study design acceptability will be evaluated through a parent-completed study specific evaluation questionnaire completed at the end of the study.
timeFrame: Day 197
Secondary outcomes:
measure: 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.
timeFrame: Day 71 and 176
measure: The frequency of adverse events as reported throughout the study will be summarized across the medicinal cannabis and placebo treatment phases.
timeFrame: Day 1 to day 197
Other outcomes:
Locations:
city: Parkville
country: Australia
facility: Royal Children's Hospital / Murdoch Children's Research Institute
state: Victoria
Study officials:
References: