Safety and Tolerability of GWP42006 in Children and Young Adults With Autism Spectrum Disorder
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 1
- 试验地点
- 1
- 主要终点
- Number of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)
研究概览
简要总结
To determine the safety and tolerability of GWP42006 (cannabidivarin, CBDV) in children and young adults with autism spectrum disorder (ASD) and to examine the effect of GWP42006 on communication, social interactions, sleep, behavior, and cognition profiles.
详细描述
This is a 52-week, open-label trial to evaluate the safety and tolerability of GWP42006. Participants who satisfy all eligibility criteria will start GWP42006 at a dose of 2.5 milligrams per kilogram per day (mg/kg/day) and titrate to a target dose of 10 mg/kg/day or 800 mg/day, whichever is smaller, during the first 4 weeks of treatment. If there is intolerance during titration, the participant may be maintained on a dose below 10 mg/kg/day. The maximum dose participants aged 6 years or older can receive will be 20 mg/kg/day or 1600 mg/day, whichever is smaller. Following the final treatment dose, participants will taper GWP42006 10% per day. The investigator will withdraw participants who fail to demonstrate any perceived benefit and may withdraw participants for whom tolerability is poor.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 4 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Autism Spectrum Disorder (ASD) as defined by the Autism Diagnostic Observation Schedule, 2nd Edition and The Diagnostic and Statistical Manual of Mental Disorders, 5th Edition
- •Intelligence quotient (IQ) of 40-120 (inclusive)
- •Participant and their caregiver are English speaking.
- •In the opinion of the investigator, the participant presents with ASD symptoms that warrant a therapeutic trial with GWP
排除标准
- •Known single gene neurogenetic disorder with high rates of epilepsy/autism (e.g., fragile X, tuberous sclerosis complex), structural brain lesion (prior stroke or hemispheric brain malformations), or history of any other epileptic encephalopathy, including infantile spasms, before the diagnosis of ASD
- •More than 2 epileptic seizures per month within the 6 months prior to screening
- •Initiation of a behavioral therapy program, new psychotropic medication, or therapeutic diet within the 2 months prior to screening, or plan to change or start any of the above during the trial
- •Presence of a significant untreated medical problem (obstructive sleep apnoea, restless legs syndrome, gastroesophageal reflux disease, etc.) which may have significant impact on sleep study measures
- •Behavioral management issues (e.g., self-injury, aggression) severe enough to be of safety concerns (to participant and/or staff)
- •Clinically significant electrocardiogram abnormality or postural drop in systolic blood pressure at screening
- •Any known or suspected hypersensitivity to cannabinoids or any of the excipients of GWP42006, such as sesame oil
- •Known history of psychiatric disorder (defined as schizophrenia, bipolar disorder, or other psychiatric disease with a known history of hallucinations or delusions)
- •History of any inborn errors of metabolism
- •Significantly impaired hepatic function at screening
- •Received an investigational product within the 3 months prior to screening
- •Participant has been taking felbamate for less than 1 year prior to screening
- •History of substance use disorders or positive drug of abuse dipstick test at screening (unless the positive result is due to a known concomitant medication)
- •Currently using or has used recreational or medicinal cannabis or cannabinoid-based medications within the 3 months prior to screening and is unwilling to abstain for the duration of the trial
- •Any history of suicidal behavior or any suicidal ideation within the month prior to or at screening
研究组 & 干预措施
GWP42006
Oral solution taken twice daily with food for 52 weeks.
干预措施: GWP42006 (Drug)
结局指标
主要结局
Number of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)
时间窗: Day 1 to Day 403
A TEAE was defined as an adverse event (AE) with an onset date on or after the first dose of GWP42006. If an AE had a partial onset date and it was unclear from the partial date (or the stop date) whether the AE started prior to or following the first dose of GWP42006, then the AE was considered a TEAE. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through to the Safety Follow-up will be presented.
次要结局
- Change from Baseline in Vineland Adaptive Behavior Scales, 3rd Edition (Vineland-3)(Baseline to End of Treatment (Day 365) or ET)
- Change from Baseline in Social Responsiveness Scale-2 (SRS-2)(Baseline to End of Treatment (Day 365) or ET)
- Change from Baseline in Children's Communication Checklist-2 (CCC-2)(Baseline to End of Treatment (Day 365) or Early Termination (ET))
- Change from Baseline in National Institutes of Health (NIH) Toolbox Cognition Battery(Baseline to End of Treatment (Day 365) or ET)
- Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ)(Baseline to End of Treatment (Day 365) or ET)
- Change from Baseline in Repetitive Behavioral Scale - Revised (RBS-R)(Baseline to End of Treatment (Day 365) or ET)
- Change from Baseline in Aberrant Behavior Checklist (ABC)(Baseline to End of Treatment (Day 365) or ET)
- Clinical Global Impressions-Improvement (CGI-Improvement)(Baseline to End of Treatment (Day 365) or ET)