CBD for Autism

Overseen By Elizabeth Wise, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: Johns Hopkins University

Must not be taking: Clobazam

Disqualifiers: Substance use disorder, Pregnancy, others

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial is testing if cannabidiol (CBD) can help with anxiety, mood issues, sleep problems, and agitation in adults with autism spectrum disorder (ASD). Participants will take CBD pills for a period of time. The study aims to see if CBD is effective and safe for these psychiatric problems. Cannabidiol (CBD) has shown promise in treating psychotic symptoms and anxiety, but there is not enough high-quality evidence for its clinical use in psychiatric disorders.

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 CBD safe for use in humans?

The provided research articles do not contain specific safety data on CBD or its use in humans. ^{1 2 3 4 5}

How does the drug CBD differ from other treatments for autism?

CBD is unique because it is being explored as a potential treatment for the core symptoms of autism, which currently have no effective standard treatment. It is derived from cannabis and is thought to help with symptoms like communication and social interaction deficits, as well as co-occurring conditions like sleep disorders and ADHD, with relatively mild side effects. ^{6 7 8 9 10}

Research Team

Elizabeth Wise, MD

Principal Investigator

Johns Hopkins University

Eligibility Criteria

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

Trial Timeline

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

Treatment Details

Interventions

• Cannabidiol (Cannabinoid)
• Placebo (Behavioural Intervention)

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.

Participant Groups

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 Canada for the following indications:

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+

Findings from Research

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]

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Considerations in assessing the abuse potential of psychedelics during drug development. [2023]

2. England pubmed.ncbi.nlm.nih.gov

A tool to utilize adverse effect profiles to identify brain-active medications for repurposing. [2018]

3. United States pubmed.ncbi.nlm.nih.gov

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

4. United States 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. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

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

6. England pubmed.ncbi.nlm.nih.gov

Cannabidiol - A new fixture in childhood neurology. [2021]

7. Switzerland 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. [2020]

8. United States pubmed.ncbi.nlm.nih.gov

Brief Report: Cannabidiol-Rich Cannabis in Children with Autism Spectrum Disorder and Severe Behavioral Problems-A Retrospective Feasibility Study. [2020]

9. England pubmed.ncbi.nlm.nih.gov

Cannabidiol as a suggested candidate for treatment of autism spectrum disorder. [2019]

10. United States pubmed.ncbi.nlm.nih.gov

Parental Perceptions and Usage of Unlicensed Cannabidiol Products in Children With Anxiety and Neurodevelopmental Disorders. [2023]