A Phase II/III Double-Blind, Randomised and Controlled-to-Open-Label Study Assessing the Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the Severity of Autism Spectrum Disorder in Young People
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 54
- 试验地点
- 2
- 主要终点
- Change in Clinical Global Impression-Severity (CGI-S)
研究概览
简要总结
This is an 18 to 54 week study assessing the efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the severity of autism spectrum disorder in young people.
详细描述
The purpose of this study is to determine the effectiveness of NTI164 in patients with ASD when treated with 20mg/kg/day for 8 - 54 weeks.
The study comprises of an 8-week double-blinded randomised controlled treatment period followed by an 8-week open-label maintenance period followed by a 2-week wash-out period. Participants who wish to continue receiving the study treatment beyond the 16 week period may do so for up to fifty-two weeks (Extension phase).
Efficacy will be measured and monitored by performing participant- and psychologist- led questionnaires specific to measuring changes in the behaviour of patients with ASD.
Safety will be measured and monitored by performing full blood examinations and liver and renal function tests throughout the study.
Additional assessments include microbiome and inflammatory marker assessments.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
盲法说明
Allocation is concealed utilising central randomisation by computer.
入排标准
- 年龄范围
- 8 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participant is aged 8 years to 17 years (inclusive)
- •Participant is at a healthy weight at the discretion of the Principal Investigator.
- •Parents or caregivers can give informed consent for participation in the trial with assent from individuals with autism.
- •Participants can comply with trial requirements.
- •According the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria the participant has a diagnosis of Level 2 or 3 Autism Spectrum Disorder (ASD) confirmed by Autism Diagnostic Observational Schedule (ADOS-2) criteria
- •All treatments including medications and therapies for ASD related symptoms must have been stable for 4 weeks before enrolment and for the duration of the trial wherever possible.
- •Participants must be able to swallow liquid.
- •Consent giver must be able to understand the requirements of the study.
排除标准
- •Current diagnosis of bipolar disorder, psychosis, schizophrenia, schizoaffective disorder, or active major depression
- •Has a diagnosis other than ASD that dominates the clinical presentation (e.g., Attention Deficit Hyperactivity Disorder [ADHD])
- •Has a degenerative condition
- •Changes in anticonvulsive therapy within the last 12 weeks
- •Taking omeprazole, lansoprazole, tolbutamide, warfarin, sirolimus, everolimus, temsirolimus, tacrolimus, clobazam, repaglinide, pioglitazone, rosiglitazone, montelukast, bupropion, or efavirenz
- •Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®, or Epidiolex®) within the 12 weeks prior to screening and is unwilling to abstain for the duration of the trial
- •Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients
- •Participant has moderately impaired hepatic function at screening, defined as serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 × upper limit of normal (ULN) or total bilirubin (TBL) > 2 × ULN. This criterion can only be confirmed once the laboratory results are available; participants enrolled into the trial who are later found to meet this criterion must be screen-failed.
- •Participant is male and fertile (i.e., after puberty unless permanently sterile by bilateral orchidectomy) unless willing to ensure that they use male contraception (condom) or remain sexually abstinent during the trial and for 12 weeks thereafter.
- •Participant is female and with childbearing potential (i.e., following menarche and until becoming postmenopausal for greater than or equal to 12 consecutive months unless permanently sterile by hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) unless willing to ensure that they use a highly effective method of birth control (e.g., hormonal contraception, intrauterine device/hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the trial and for 12 weeks thereafter.
- •Female participant who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the trial or within 12 weeks thereafter.
- •Participant had brain surgery or traumatic brain injury within 1 year of screening.
- •Participant has any other significant disease or disorder which, in the opinion of the investigator, may either put the participant, other participants, or site staff at risk because of participation in the trial, may influence the result of the trial, or may affect the participant's ability to take part in the trial.
- •Any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the trial
- •Any history of suicidal behaviour (lifelong) or any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) in the last 4 weeks or at screening or randomization
- •Participant has donated blood during the past 12 weeks and is unwilling to abstain from donation of blood during the trial.
- •Participant has any known or suspected history of alcohol or substance abuse or positive drugs of abuse test at screening (not justified by a known concurrent medication).
- •Participant has previously been enrolled into this trial.
- •Participant has plans to travel outside their country of residence during the trial, unless the participant has confirmation that the product is permitted in the destination country/state.
研究组 & 干预措施
Placebo
Randomised Controlled Phase:
Part A: 5mg/kg, 10mg/kg, 15mg/kg, 20mg/kg (1 week each, total duration = 4 weeks) Part B: 20mg/kg or maximum tolerated dose (total duration = 4 weeks).
干预措施: NTI164 (Drug)
NTI164
Full-Spectrum Medicinal Cannabis Plant Extract with less than 0.08% THC (NTI164)
Randomised Controlled Phase:
Part A: 5mg/kg, 10mg/kg, 15mg/kg, 20mg/kg (1 week each, total duration = 4 weeks) Part B: 20mg/kg or maximum tolerated dose (total duration = 4 weeks).
Open-Label Phase 20mg/kg or maximum tolerated dose (total duration = 8 weeks).
Extension Phase 20mg/kg or maximum tolerated dose (total duration = 36 weeks).
干预措施: NTI164 (Drug)
结局指标
主要结局
Change in Clinical Global Impression-Severity (CGI-S)
时间窗: Baseline, Week 8.
Reflects clinician's impression of severity of illness on a 7-point scale ranging from 1=not at all to 7=among the most extremely ill.
次要结局
- Vineland Adaptive Behaviour Scales, Third Edition(Baseline, Weeks 16, 28, 40 & 52)
- Social Responsiveness Scale, 2nd Editions (SRS-2)(Baseline, Weeks 16, 28, 40 & 52)
- Clinical Global Impression Scale - Improvement (CGI-I)(Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52)
- Anxiety, Depression and Mood Scale (ADAMS)(Baseline, Weeks 16, 28, 40 & 52)
- Sleep Disturbance Scale for Children (SDSC)(Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52)
- Anxiety Scale for Children - Autism Spectrum Disorder(Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52)
- Caregiver Global Impression of Change in Attention (CGI-CA)(Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52)
- Caregiver Global Impression of Change (CGI-C) Target Behaviour(Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52)