A Pilot Randomized Placebo-controlled Crossover Trial of Medicinal Cannabis (MC) in Adolescents with Tourette Syndrome (TS)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Rate of study participant recruitment, calculated as the time required to reach a sample size of 10.
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 12 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
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).
干预措施: Placebo (Drug)
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).
干预措施: Placebo (Drug)
结局指标
主要结局
Rate of study participant recruitment, calculated as the time required to reach a sample size of 10.
时间窗: From the date of pre-screening the first participant until the tenth participant is randomized, up to 2 years.
The rate of recruitment will be calculated as the number of months from the date of commencing recruitment to the date of randomizing the tenth participant.
Self-report questionnaire completion, calculated as the proportion of adolescent self-report questionnaires completed across the study sample.
时间窗: Screening to day 169 (final study visit)
The number of study self-report questionnaires completed by all participants will be calculated as a proportion of the total possible questionnaires requiring completion in accordance with the study protocol.
Study visit attendance, calculated as the proportion of visits completed across the study sample.
时间窗: Screening to day 169 (final study visit)
The number of study visits attended by all participants will be calculated as a proportion of the total possible visits in accordance with the study protocol.
Study medication tolerability, as indicated by the proportion of participants who tolerate the protocol dosing schedule.
时间窗: Day 1 to day 176 (end of treatment period 2)
The number of participants who adhere to the protocol dosing schedule without medication related protocol deviations, treatment discontinuations or dose modifications will be calculated as a proportion of the total sample for each treatment condition (medicinal cannabis or placebo).
Parent questionnaire completion, calculated as the proportion of parent-report questionnaires completed across the study sample.
时间窗: Screening to day 169 (final study visit)
The number of study questionnaires completed by all parents will be calculated as a proportion of the total possible questionnaires requiring completion in accordance with the study protocol.
Participant withdrawal rate, calculated as the number of participants who withdraw from the trial as a proportion of the total number of participants randomized.
时间窗: Day 1 to day 176 (end of treatment period 2)
The number of participants who withdraw from the trial will be calculated as a proportion of the total number of participants randomized.
Participant adherence to the study medication dosing schedule, calculated as the proportion of participants who demonstrate acceptable medication compliance.
时间窗: Day 78 (end of treatment period 1) and day 176 (end of treatment period 2)
Medication compliance will be assessed through pharmacy calculations from returned bottle volumes. Acceptable compliance will fall within the range of 80-120%. The number of participants with acceptable medication compliance will be reported as a proportion of the total sample randomized.
Blood test completion, calculated as the proportion of blood tests completed across the study sample.
时间窗: Screening to day 169 (final study visit)
The number of study blood tests completed by all participants will be calculated as a proportion of the total possible blood tests in accordance with the study protocol.
Study design acceptability will be evaluated through a parent-completed study specific evaluation questionnaire completed at the end of the study.
时间窗: Day 197
Study design acceptability will be assessed using an evaluation questionnaire developed specifically for this study, which uses Likert scales to assess satisfaction with recruitment, medication tolerability, frequency of study visits, burden of completing questionnaires, and overall study quality. Parents will complete this questionnaire at the end of their study participation (day 197). Data will be reported for each item individually, as the proportion of parents who responded positively on the Likert scale, where higher scores indicate more favorable responses.
次要结局
- The frequency of adverse events as reported throughout the study will be summarized across the medicinal cannabis and placebo treatment phases.(Day 1 to day 197)
- 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.(Day 71 and 176)