CompletedPhase 3ACTRN12621001659897

A pilot randomised controlled trial testing the efficacy of cannabidiol for anxiety in Tourette Syndrome

Sponsor

Children's Health Queensland Hospital and Health Service

Enrollment

20 participants

Start Date

Mar 25, 2022

Study Type

Interventional

Conditions

Anxiety Tourette Syndrome

Summary

This trial will identify the efficacy of CBD for the treatment of anxiety in children and adolescents with Tourette Syndrome. Forty children and adolescents aged 6 to 17 years will be recruited to a 32-week randomised, double-blind, placebo controlled cross-over trial. Participants will be randomised on a 1:1 basis to either the active group (CBD) or control group (placebo). Following 14 weeks of the trial participants will then cross-over to the alternative group for comparison. It is proposed that while participants are in the active group their levels of anxiety will be significantly lower than when they are in the placebo group. Similarly, it is proposed that participants in the active group will also have significantly less tics, lower levels of depression, decreased obsessive-compulsive behaviours and improved quality of life, sleep and global symptomology.

Eligibility

Sex: Both males and femalesMin Age: 6 YearssMax Age: 17 Yearss

Inclusion Criteria5

Male or female children/adolescents aged 6-17 years old.
Participants must have a diagnosis of Tourette Syndrome [confirmed by psychiatric diagnosis], and who have either (1) trialled a course of pharmaceutical (e.g. Clonidine), and psychological (e.g. cognitive behavioural therapy) treatment unsuccessfully; or, (2) who have significant contra-indications to standard methods of treatment
Participants must be experiencing severe symptoms of anxiety as assessed by the PI at screening
If participants are receiving non-pharmacological educational, behavioural, and/or dietary interventions, they must be stable and have been doing so for 2 months prior to screening.
Participants must be willing to continue to be reviewed by their primary treating doctor/psychiatrist/paediatrician during the trial.

Exclusion Criteria10

Use of any cannabis-based product within a month prior to the screening visit (confirmed via urine test at screening).
Planned use of any cannabis-based product (or other investigational drug) other than that offered in this trial for the planned duration of this trial.
History of treatment for, or evidence of, drug abuse within the past year.
History of suicidal behaviour and active intent.
Participants with pre-existing cardiac conditions or abnormalities.
Known allergy to CBD or any other cannabinoid and the excipients (ethanol, sucralose, strawberry flavour, sesame oil).
Participants currently using strong CYP 3A4 inhibitors/inducers or sensitive substrates which may interact with CBD
Any clinically significant condition or abnormal findings at the Screening Visit that would, in the opinion of the PI, preclude study participation or interfere with the evaluation of the study treatment.
Participants experiencing acute or progressive neurological disease, psychiatric disorder or severe cognitive abnormalities that are likely to require changes in drug therapy, interfere with the objectives of the trial, or interfere with the ability to adhere to protocol requirements.
Females who are pregnant, nursing or are planning a pregnancy. Cannabidiol is contraindicated in pregnancy. Females of childbearing potential will require a negative serum pregnancy test before participating in the study. Females of childbearing potential must also be willing to use a suitable contraceptive during the trial if they plan on being sexually active. Urine pregnancy tests will be conducted and must remain negative to be permitted to continue the trial.

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Interventions

This is a randomised, double-blind, placebo-controlled cross-over trial to assess the efficacy of Cannabidiol (CBD) administered as Epidiolex. Epidiolex is a purified cannabidiol oral solution that will be administered orally or via gastrostomy and nasogastric feeding tubes. All participants will undergo a screening process. Eligible participants will be randomised to begin either 16-weeks of treatment with CBD or 16-weeks of treatment with placebo. Each treatment block includes 6 weeks of titration, 8 weeks on the maintenance dose and 2 weeks of drug tapering. Following the initial 16-weeks of either treatment or placebo, patients will immediately cross-over and begin a further 16-weeks of the alternative treatment. Allocation to treatment group will be randomised and blinded for both participants and study personnel. Treatment group A (Epidiolex) Parents/caregivers will administer the study drug orally/via gastrostomy/via nasogastric tube in two divided doses (12 hours +/- 2 hours) daily. Dosage will be titrated over 6 weeks until maximum or best tolerated dose is reached. Maximum or best tolerated dose will be maintained for 8 weeks. Participants are then titrated down over a two week period, before crossing over to the alternative treatment arm. Dosing schedule: Week 1-2: 2.5mg/kg/day via two divided doses Week 3-4: 5mg/kg/day via two divided doses Week 5-6: 10mg/kg/day via two divided doses Week 7-14: 15mg/kg/day Week 15-16: 5mg/kg/day (Week 15); 2.5mg/kg/day (Week 16) Four weeks after final dose, participants will be followed up in clinic prior to discharge from the study. Compliance with study medication will be reviewed at each "in-clinic" appointment. Pharmacy staff will calculate the expected number of bottles (based on participants weight and expected titration schedule) required between clinic visit time points and dispense to the participants accordingly. Participants will be required to return all used and/or any unused bottles of medication to the study site pharmacy where clinical trial pharmacists will complete accountability and adherence calculations. This information will be forwarded to the PI and/or PO for noting. A new prescription for the study medication will be provided by the PI to the clinical trial pharmacists who will dispense a new supply of study medication to see participants through until their next in-clinic appointment.