A Phase 2, Placebo-Controlled, Randomized, Double-Blind Study to Assess the Safety, Tolerability and Efficacy of Terpenes-enriched Cannabis Oil T1/C28, Administered to Pediatric Subjects With Autism Spectrum Disorder (ASD)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 78
- 试验地点
- 1
- 主要终点
- Change From Baseline in Aberrant Behavior Checklist-Community (ABC-C): Irritability Subscale raw score (ABC-I)
研究概览
简要总结
The goal of this clinical trial is to determine whether cannabidiol (CBD, 28%) combined with terpenes and a small amount of THC (1%) can help reduce symptoms of autism, and to evaluate the safety of this treatment.
The main questions are:
- Does this treatment improve behavioral challenges in children with autism?
- Does this treatment improve social difficulties in children with autism?
What will happen in the study:
- Participants take either the study treatment or a placebo (a look-alike substance with no active drug) every day for 2 months.
- After 2 months, all participants receive the study treatment or a similar treatment without THC for another 2 months.
- Participants come to the clinic once every 2 months for checkups and tests.
详细描述
Autism spectrum disorder (ASD) is a condition that affects communication, social interaction, and behavior. Current medications do not treat the core symptoms of autism, and the drugs sometimes prescribed (such as antipsychotics for irritability) can cause significant side effects.
Cannabidiol (CBD) is a natural, non-psychoactive compound from the cannabis plant that may reduce brain overactivity and inflammation. Tetrahydrocannabinol (THC), the psychoactive component of cannabis, acts on the endocannabinoid system, which is thought to function differently in people with autism. Research suggests that CBD combined with very small amounts of THC may improve behavior and social functioning. Other plant compounds called terpenes may enhance the effects of CBD and THC, even at low doses.
This study tests whether a CBD oil enriched with terpenes and a very small amount of THC is safe and effective for children with autism. Seventy-eight children, ages 4-13, will participate. Half will receive the study oil and half will receive a placebo (an inactive oil that looks the same) for 8 weeks. Afterward, all participants will receive an active treatment for another 8 weeks.
The study evaluates whether the treatment improves behavior, social skills, and quality of life. Safety is monitored through regular clinic visits, questionnaires, physical exams, and blood tests.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 13 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children aged 4 to 12 years (after the 4th birthday and before the 13th).
- •Diagnosis of autism spectrum disorder (ASD) according to DSM-5, confirmed by Childhood Autism Rating Scale-Second Edition (CARS-2).
- •Moderate or greater ASD-associated symptoms, defined as a rating of ≥4 ("moderate" or higher) on the Overall Function Clinical Global Impression-Severity (CGI-S).
- •Aberrant Behavior Checklist-Irritability subscale (ABC-I) score ≥
- •Social Responsiveness Scale-Second Edition (SRS-2) total T score ≥66.
排除标准
- •Body weight <12.5 kg or ≥57.5 kg.
- •Current or past diagnosis of heart failure, drug addiction, schizophrenia, psychosis, bipolar disorder, post-traumatic stress disorder (PTSD), or major depressive disorder (MDD), or diagnosis of schizophrenia in a first-degree relative.
- •Seizure or change in antiepileptic medications within 4 months prior to randomization.
- •Clinically significant abnormalities on physical examination or laboratory testing, including impairment in cardiac, hepatic, or renal function.
- •Change in pharmacological or behavioral treatment, or change in home or school environment (other than school holidays), within 4 weeks prior to randomization or planned during the study.
- •Cannabinoid treatment within 4 weeks prior to randomization.
- •Predicted poor compliance with study procedures (e.g., blood tests).
- •Concurrent use of opiates or alcohol.
研究组 & 干预措施
Terpenes-Enriched CBD-Predominant Oil
Oral cannabidiol (CBD), tetrahydrocannabinol (THC; 1/28 of the CBD dose), and terpenes, administered for 8 weeks as an add-on to existing treatments. After the first 8 weeks, participants will continue with the same treatment for an additional 8 weeks.
干预措施: Terpenes-Enriched CBD-Predominant Oil (Drug)
Placebo
Oral olive oil with flavoring to mimic the texture and taste of the study drug, given for 8 weeks. After the first 8 weeks, participants will switch to terpenes-enriched CBD oil without THC for another 8 weeks.
干预措施: Placebo (Drug)
Placebo
Oral olive oil with flavoring to mimic the texture and taste of the study drug, given for 8 weeks. After the first 8 weeks, participants will switch to terpenes-enriched CBD oil without THC for another 8 weeks.
干预措施: Terpenes-Enriched CBD Oil (THC-Free) (Drug)
结局指标
主要结局
Change From Baseline in Aberrant Behavior Checklist-Community (ABC-C): Irritability Subscale raw score (ABC-I)
时间窗: Baseline to Week 8
The ABC-C is a caregiver-completed questionnaire with 58 items, divided into five subscales. The ABC-I (Irritability) subscale includes 15 items that assess emotional and behavioral symptoms of ASD, such as aggression toward others, deliberate self-injury, temper tantrums, depressed mood, and rapidly shifting moods. Scores on this subscale range from 0 to 45, with higher scores indicating greater severity. Change from baseline to Week 8 is reported, with lower scores reflecting clinical improvement.
次要结局
- Caregiver Global Impression of Change (CGIC or CaGC)(Weeks 8 and 17)
- Change From Baseline in Vineland™-III Adaptive Behavior Scales (VABS3) Socialization Domain Standard Score(Baseline to Week 8)
- Change From Baseline in Social Responsiveness Scale-2nd edition (SRS-II) total raw score(Baseline, Weeks 8 and 17)
- Number of Participants Reporting Treatment-emergent Adverse Events (TEAEs)(Baseline to Week 17)
- Clinical Global Impressions-Improvement (CGI-I) Score(Weeks 8 and 17)
- Number of Participants With Clinically Significant Abnormal Laboratory Values(Week 8)
- Change From Baseline in Body Mass Index (BMI)(Baseline, Weeks 8 and 17)
研究者
Adi Aran
Director, Neuropediatric unit, SZMC
Shaare Zedek Medical Center