Placebo-Controlled Study of Terpenes-Enriched Cannabis Oil T1/C28 for Children With Autism

Official title

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)

Overview

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: 1. Does this treatment improve behavioral challenges in children with autism? 2. Does this treatment improve social difficulties in children with autism? What will happen in the study: 1. Participants take either the study treatment or a placebo (a look-alike substance with no active drug) every day for 2 months. 2. After 2 months, all participants receive the study treatment or a similar treatment without THC for another 2 months. 3. Participants come to the clinic once every 2 months for checkups and tests.

Detailed description

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.

Interventions

• Drug Terpenes-Enriched CBD-Predominant Oil
  Oral cannabidiol (CBD; 7.2 mg/kg/day), tetrahydrocannabinol (THC; 0.257 mg/kg/day, equivalent to 1:28 of the CBD dose), and terpenes (0.5 mg/kg/day), administered in two daily doses. The formulation is an olive oil-based solution (CBD/THC: 280/10 mg per g) produced by Bazelet Group, Israel.
• Drug Placebo
  Oral olive oil with added flavors to mimic the appearance, texture, and taste of the study drug, administered in two daily doses.
• Drug Terpenes-Enriched CBD Oil (THC-Free)
  Oral cannabidiol (CBD; 7.2 mg/kg/day) and terpenes (0.5 mg/kg/day), administered in two daily doses. The formulation is an olive oil-based solution (CBD- 280 mg per g) produced by Bazelet Group, Israel.

Primary outcome measures

• Change From Baseline in Aberrant Behavior Checklist-Community (ABC-C): Irritability Subscale raw score (ABC-I) [Time frame: Baseline to Week 8]

Secondary outcome measures (7)

• Change From Baseline in Vineland™-III Adaptive Behavior Scales (VABS3) Socialization Domain Standard Score [Time frame: Baseline to Week 8]
• Change From Baseline in Social Responsiveness Scale-2nd edition (SRS-II) total raw score [Time frame: Baseline, Weeks 8 and 17]
• Number of Participants Reporting Treatment-emergent Adverse Events (TEAEs) [Time frame: Baseline to Week 17]
• Number of Participants With Clinically Significant Abnormal Laboratory Values [Time frame: Week 8]
• Change From Baseline in Body Mass Index (BMI) [Time frame: Baseline, Weeks 8 and 17]
• Clinical Global Impressions-Improvement (CGI-I) Score [Time frame: Weeks 8 and 17]
• Caregiver Global Impression of Change (CGIC or CaGC) [Time frame: Weeks 8 and 17]

Eligibility criteria

Inclusion criteria

• 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 ≥18.
• Social Responsiveness Scale-Second Edition (SRS-2) total T score ≥66.

Exclusion criteria

• 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

Israel · 1 center

• Shaare Zedek Medical Center — Jerusalem

Identifiers

NCT: NCT07199218 · 0336-24-SZMC · MOH_2025-04-21_014042

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗