CBD for Autism

(CASCADE Trial)

Overseen By Rebecca Rochowiak, MA

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Colorado, Denver

Must be taking: Psychotropics, AEDs

Must not be taking: Clobazam, Felbamate

Disqualifiers: Pregnancy, THC use, Neurological disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 27 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of cannabidiol (CBD) in assisting children and adolescents with autism spectrum disorder (ASD). Participants will receive either oral cannabidiol 100mg/mL or a placebo (a non-active substance) to determine if CBD can improve symptoms. The trial consists of three groups, with some receiving CBD throughout and others alternating between CBD and placebo. Eligible participants must have a documented ASD diagnosis and face moderate to severe daily challenges due to the condition. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group, providing participants an opportunity to contribute to significant findings.

Will I have to stop taking my current medications?

You can continue taking your current medications if they are stable and include no more than two psychotropic medications or anti-epileptic drugs. You must maintain the same medication regimen throughout the study.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that cannabidiol (CBD) is generally safe for children with autism. Studies found that children tolerated CBD well, experiencing no major side effects compared to a placebo. However, more research is needed to understand its long-term effects.

Parents have observed improvements in autism symptoms with CBD treatment, but they also stress the need for further research on its long-term safety. Overall, current studies suggest that CBD is safe for short-term use and is not associated with serious side effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Unlike the standard options for autism, which typically include behavioral therapy and medications like antipsychotics and stimulants, cannabidiol (CBD) offers a novel approach. Researchers are excited about CBD because it is derived from cannabis but doesn't produce a high, making it a promising option for reducing symptoms without the side effects associated with traditional medications. CBD's mechanism of action involves interacting with the endocannabinoid system, which may help modulate mood and behavior in a way current treatments do not. This potential for a more natural and possibly safer alternative has generated significant interest in the medical community.

What evidence suggests that this trial's treatments could be effective for autism?

Research has shown that cannabidiol (CBD) might help with autism symptoms. Some studies indicate that CBD can greatly improve social skills, reduce anxiety, and enhance movement skills involving both the brain and muscles. Evidence also suggests that CBD can safely improve severe autism symptoms. In certain trials, children and teens with autism behaved better when taking CBD compared to a placebo. In this trial, participants will be assigned to different treatment arms, with some receiving CBD for varying durations and others receiving a placebo. While more research is needed, these findings suggest that CBD could be a promising treatment for challenges related to autism. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

This trial is for children and adolescents aged 5-17 with Autism Spectrum Disorder (ASD). They must be on a stable regimen of no more than two psychotropic or anti-epileptic drugs, if applicable, and not planning to change treatments during the study. Participants need a BMI between 12-32 kg/m2 and have been diagnosed by a professional. Females who can bear children must test negative for pregnancy.

Inclusion Criteria

Parents/caregivers need to be able to read and answer questions and forms in English.

If you take medication for mental health, you need to have been on the same medication for at least 4 weeks before the study starts, and you can't change it during the study.

If you are getting non-medication treatments or therapies, they must be stable for at least 2 months before the screening. Changes during school breaks are okay.

Exclusion Criteria

Plan to initiate or change pharmacologic or non-pharmacologic interventions during the course of the study

You have a suspected or confirmed heart or blood vessel problem.

Adolescent females who are pregnant, nursing, or planning a pregnancy. Females of childbearing potential and male patients with a partner of childbearing potential who are unwilling or unable to use standard acceptable methods of contraception (including abstinence, hormonal contraception, diaphragm, cervical cap, vaginal sponge, condom, or intrauterine device) for the duration of the study and for 1 month after the last dose of study medication

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either cannabidiol or placebo for 12 to 27 weeks, depending on the group assignment

27 weeks

Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Oral cannabidiol 100mg/mL
• Placebo

Trial Overview The CASCADE trial tests oral cannabidiol (CBD) against a placebo in kids with ASD. It's randomized and controlled, meaning some get CBD while others get an inactive substance without knowing which one they receive. All participants will eventually receive CBD for at least 12 weeks during the study.

How Is the Trial Designed?

Group I: Arm 3: 27 weeks of study drug Experimental Treatment 1 Intervention

Subjects in this group will receive cannabidiol throughout the entire 27 weeks of treatment.

Group II: Arm 3: 15 weeks of placebo then 12 weeks of study drug Experimental Treatment 2 Interventions

Subjects in this group will receive placebo for the first 15 weeks of the study and cannabidiol for the last 12 weeks.

Group III: Arm 1: 12 weeks of study drug then 15 weeks of placebo Experimental Treatment 2 Interventions

Subjects in this group will receive cannabidiol for the first 12 weeks of the study and placebo for the last 15 weeks.

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials

1,842

Recruited

3,028,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12005571/

Efficacy and Safety of Cannabinoids for Autism Spectrum ... Regarding core ASD symptoms and behavioral outcomes, cannabinoids demonstrated greater efficacy compared to placebo in some studies. However, ...

2. europsy.net europsy.net/app/uploads/2025/04/Cannabidiol-Therapy-Could-Reduce-Symptoms-in-Autistic-Children-and-Teenagers.pdf

Cannabidiol Therapy Could Reduce Symptoms in Autistic ... We included randomized, placebo-controlled trials on the efficacy or safety of CBD cannabis extracts in children and adolescents with ASD. For ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04517799

Trial of Cannabidiol to Treat Severe Behavior Problems in ... If CBD is found to be safe and effective in treating the behavioral problems associated with autism, this would be a major new tool in the treatment of those ...

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0091305725000188

Purified cannabidiol leads to improvement of severe ... Our results suggest that treatment with purified CBD is effective and safe and could benefit patients with severe ASD by improving some of the core symptoms.

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11332686/

randomized, double-blind, and placebo-controlled clinical trial Based on the results obtained, it can be concluded that CBD-rich cannabis extract showed significant improvement in social interaction, anxiety, and psychomotor ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04745026

NCT04745026 | Trial to Investigate the Safety and Efficacy ... Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder.

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6333745/

Oral Cannabidiol Use in Children With Autism Spectrum ... Conclusion: Parents' reports suggest that cannabidiol may improve ASD comorbidity symptoms; however, the long-term effects should be evaluated ...

8. medrxiv.org medrxiv.org/content/10.1101/2023.12.05.23299505v1.full-text

Safety and efficacy of orally administered full-spectrum ... In addition, two randomised controlled trials have shown that cannabinoid treatment, compared to placebo in children, was safe with acceptable ...

9. scielo.br scielo.br/j/trends/a/TVWvDrn75t5vHNbfYSvgzTz/?lang=en

Evaluation of the efficacy and safety of cannabidiol-rich ... Conclusion CBD-rich cannabis extract was found to improve one of the diagnostic criteria for ASD (social interaction), as well as features that often co-exist ...