CBD for Autism

Overseen By Elizabeth Wise, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Johns Hopkins University

Must not be taking: Clobazam

Disqualifiers: Substance use disorder, Pregnancy, others

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 8

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether CBD, a compound from cannabis, can alleviate anxiety, mood issues, sleep problems, and agitation in adults with autism. Participants will take a CBD pill for six weeks, a placebo for six weeks, and have a two-week break. Eligible participants must have autism and experience significant mood, sleep, or behavioral issues that affect daily life. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are taking medications that interact with CBD, like clobazam.

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

Research has shown that CBD (cannabidiol) might be safe for people with autism spectrum disorder (ASD). In past studies, many participants using CBD exhibited better behavior without serious side effects. For example, one study found that purified CBD improved symptoms in individuals with severe ASD and was well-tolerated.

Another study focused on children and teenagers with ASD and found that CBD extracts did not cause major issues. Participants in these studies typically reported mild side effects, such as fatigue.

This trial is in the early stages, so there is limited information on safety for adults with ASD. However, since CBD is already used for other conditions, it suggests potential safety. Regular check-ins during the trial will help ensure participants' well-being. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for autism, which often involve behavioral therapy and medications like antipsychotics, cannabidiol (CBD) offers a new approach by harnessing compounds derived from cannabis plants. Researchers are excited about CBD because it targets the body's endocannabinoid system, which may help regulate mood and behavior without the side effects typically associated with traditional medications. Additionally, CBD is non-psychoactive, meaning it doesn't produce the "high" that is commonly associated with cannabis, making it a safer option for children and young adults.

What evidence suggests that CBD might be an effective treatment for autism?

Research has shown that cannabidiol (CBD), which participants in this trial may receive, might help improve symptoms in people with autism spectrum disorder (ASD). Some studies found that CBD can work better than a placebo in easing main ASD symptoms like anxiety and mood issues. Participants in those studies noticed improvements in difficult behaviors and daily activities. Other research suggests that purified CBD is safe and helpful, possibly reducing severe ASD symptoms. While these results are promising, more studies are needed to fully understand how CBD affects adults with ASD. ^{1 2 3 5 6}

Who Is on the Research Team?

Elizabeth Wise, MD

Principal Investigator

Johns Hopkins University

Are You a Good Fit for This Trial?

Adults with Autism Spectrum Disorder (ASD) experiencing mood disorders, sleep disturbances, or agitation can join this trial. They must meet DSM-5 criteria for ASD and have symptoms affecting their life quality. Excluded are those with substance abuse history, positive THC tests, pregnancy or lactation plans soon after the trial, unstable liver disease, or on certain medications.

Inclusion Criteria

ASD based on Diagnostic Statistical Manual 5 (DSM-5) criteria

Presence of a significant mood disorder, sleep disturbance, or exhibit agitation, aggression, or other aberrant behavior that is interfering with function and quality of life, as determined by their psychiatric interview

Exclusion Criteria

Positive urine tetrahydrocannabinol screen at onset of study

History of alcohol or substance use disorder

Individuals who are pregnant, lactating, or planning pregnancy during or within three months of completing the trial

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment - Cannabidiol

Participants receive cannabidiol, starting at 100 mg twice daily, increased to 200 mg twice daily by week 3, for six weeks

6 weeks

4 visits (in-person), 2 visits (telephone)

Washout

Participants receive no drug/placebo for two weeks to clear the effects of the previous treatment

2 weeks

Treatment - Placebo

Participants receive placebo, an inactive pill, for six weeks

6 weeks

4 visits (in-person), 2 visits (telephone)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Cannabidiol
• Placebo

Trial Overview The study is testing Cannabidiol (CBD)'s effectiveness in treating psychiatric issues in adults with ASD over a 14-week period. Participants will take CBD pills for six weeks, no treatment for two weeks, then placebo pills for another six weeks while undergoing regular assessments.

How Is the Trial Designed?

Group I: Cannabidiol Experimental Treatment 1 Intervention

Participants will receive cannabidiol, starting at 100 mg twice daily, and increased to 200 mg twice daily by week 3. This arm will last six weeks.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive six weeks of placebo.

Cannabidiol is already approved in United States, European Union, Canada for the following indications:

Approved in United States as Epidiolex for:

• Seizures associated with Lennox-Gastaut syndrome
• Seizures associated with Dravet syndrome
• Seizures associated with tuberous sclerosis complex

Approved in European Union as Epidiolex for:

• Seizures associated with Lennox-Gastaut syndrome
• Seizures associated with Dravet syndrome
• Seizures associated with tuberous sclerosis complex

Approved in Canada as Epidiolex for:

• Seizures associated with Lennox-Gastaut syndrome
• Seizures associated with Dravet syndrome

Who Is Running the Clinical Trial?

Johns Hopkins University

Lead Sponsor

Trials

2,366

Recruited

15,160,000+

Charlotte's Web, Inc

Industry Sponsor

Trials

2

Recruited

90+

Canopy Growth Corporation

Industry Sponsor

Trials

15

Recruited

1,200+

Published Research Related to This Trial

Classic psychedelics, which are currently classified as Schedule I drugs due to perceived high abuse potential, have not been systematically evaluated for their abuse potential using modern research methods since their classification in 1970.

This paper highlights the need for updated scientific assessments of classic psychedelics to support potential rescheduling if they gain FDA approval for therapeutic use, reflecting a shift in understanding their safety and efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Considerations in assessing the abuse potential of psychedelics during drug development. Calderon, SN., Bonson, KR., Reissig, CJ., et al.[2023]

Analyzing the adverse effect profiles of FDA-approved drugs can help identify which compounds are more likely to penetrate the blood-brain barrier, making them suitable candidates for central nervous system (CNS) applications.

Models that included adverse effect similarity data were better at distinguishing between drugs that can and cannot penetrate the brain, suggesting that this approach could speed up the process of repurposing existing medications for new CNS-related treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A tool to utilize adverse effect profiles to identify brain-active medications for repurposing. McCoy, TH., Perlis, RH.[2018]

Cannabidiol (CBD) is being explored as a potential treatment for Autism Spectrum Disorder (ASD) and its co-morbidities, but current evidence does not convincingly demonstrate its efficacy or safety in young patients with ASD.

Despite the growing interest in cannabinoids for treating ASD, there is a lack of robust pre-clinical and clinical data to support their use as effective treatments for the core symptoms of the disorder.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cannabidiol as a suggested candidate for treatment of autism spectrum disorder. Poleg, S., Golubchik, P., Offen, D., et al.[2019]

About 31.3% of parents surveyed reported giving unlicensed cannabidiol (CBD) products to their children diagnosed with ADHD, autism spectrum disorder (ASD), or generalized anxiety disorder (GAD), indicating a significant prevalence of CBD use in this population.

Parents who used CBD products generally perceived them as safer and felt more community support for their use, suggesting a need for healthcare providers to discuss the safety and implications of CBD use with families.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parental Perceptions and Usage of Unlicensed Cannabidiol Products in Children With Anxiety and Neurodevelopmental Disorders. Schwaller, K., Krauss, Z., Chen, AMH., et al.[2023]

The permitted daily exposure (PDE) for flutamide, an androgen receptor blocker, was determined to be 0.025 mg/d based on toxicity studies, indicating that this dose is unlikely to pose any risk to humans.

The analysis of various toxicity studies converged on a no adverse effect level (NOAEL) of 1 mg/kg/d, supporting the safety of flutamide at low exposure levels, while nonmonotonic dose response effects were not supported by the literature.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Permitted Daily Exposure of the Androgen Receptor Antagonist Flutamide. Zacharia, LC.[2018]

In a survey of 20 companies, it was found that there are four different approaches to reporting toxic/adverse dose levels in first-in-human (FIH)-enabling toxicology studies for anticancer drugs, with 45% of companies reporting the highest non-severely toxic dose (HNSTD) or the dose severely toxic to 10% (STD10).

The study highlights the importance of defining a consistent reporting approach before starting toxicology studies, as this can impact regulatory acceptance and the safety assessment of new anticancer pharmaceuticals.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Toxicologic Pathology Forum: Opinion on Approaches for Reporting Toxic and Adverse Dose Levels in Nonclinical Toxicology Studies Supporting the Development of Anticancer Pharmaceuticals. Hukkanen, RR., Moriyama, T., Patrick, DJ., et al.[2023]

High-Throughput Screening (HTS) of approximately 8,300 chemicals revealed evidence of non-monotonic dose-response (NMDR) relationships, which can complicate the assessment of chemical safety and efficacy, particularly for androgen receptor (AR) agonism.

The study identified NMDR in several compounds, including known AR antagonists and endocrine disruptors, highlighting the need for careful interpretation of HTS data to avoid misclassifying chemical activity based on traditional monotonic dose-response assumptions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Examining the evidence of non-monotonic dose-response in Androgen Receptor agonism high-throughput screening assay. Klimenko, K.[2021]

Cannabidiol (CBD) has gained attention for its potential use in treating pediatric neurological disorders, especially following its recent FDA approval, which emphasizes the need for a better understanding of its mechanisms and safety.

The article discusses both the current applications and ongoing controversies surrounding CBD use in children, highlighting the importance of evaluating long-term effects and appropriate indications for its use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cannabidiol - A new fixture in childhood neurology. Anderson, CL., Carney, PR.[2021]

In an observational study of 18 autistic patients treated with a CBD-enriched Cannabis sativa extract, most showed significant improvements in various symptoms of Autism Spectrum Disorders after 6-9 months, particularly in areas like seizures, attention deficit/hyperactivity disorder, and communication.

Despite some patients experiencing mild adverse effects leading to discontinuation, the majority of those who adhered to the treatment reported improvements, with many able to reduce or stop other medications while maintaining these benefits.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of CBD-Enriched Cannabis sativa Extract on Autism Spectrum Disorder Symptoms: An Observational Study of 18 Participants Undergoing Compassionate Use. Fleury-Teixeira, P., Caixeta, FV., Ramires da Silva, LC., et al.[2020]

In a retrospective study of 60 children with autism spectrum disorder (ASD), cannabidiol-rich cannabis treatment led to significant improvements in behavioral problems, with 61% of participants showing much or very much improvement.

While the treatment was generally well-tolerated, some adverse effects were noted, including sleep disturbances (14%) and irritability (9%), highlighting the need for careful monitoring during treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Brief Report: Cannabidiol-Rich Cannabis in Children with Autism Spectrum Disorder and Severe Behavioral Problems-A Retrospective Feasibility Study. Aran, A., Cassuto, H., Lubotzky, A., et al.[2020]

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. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11595093/

The Evolving Role of Cannabidiol-Rich Cannabis in People ... Studies indicate that treatment with cannabidiol (CBD)-rich cannabis may possess the potential to improve fundamental ASD symptoms as well as ...

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. 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 ...

5. 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.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT07199218

Placebo-Controlled Study of Terpenes-Enriched Cannabis ... 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, ...