Cannabidiol for Autism

Recruiting at 1 trial location

Overseen By Doris Trauner, MD

Age: < 18

Sex: Male

Trial Phase: Phase 2

Sponsor: University of California, San Diego

Must not be taking: Anticonvulsants, CBD

Disqualifiers: Epilepsy, Genetic conditions, Cardiac disease, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 5

42 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing whether a medicine called cannabidiol (CBD) can help boys aged 7-14 with autism who have severe behavior problems. CBD, which comes from the cannabis plant but doesn't make you feel 'high,' might help by calming brain activity. The goal is to see if this treatment can improve their behavior and quality of life. Cannabidiol (CBD) has been reported to be safe and well-tolerated and has shown potential in managing symptoms of autism.

Will I have to stop taking my current medications?

If your child is taking anticonvulsant medications like clobazam or valproic acid, they cannot participate in the trial. If your child is taking CBD from another source, you must stop the treatment for at least 4 weeks before joining the study.

What evidence supports the effectiveness of the drug Epidiolex (Cannabidiol) for treating autism?

Some studies suggest that cannabidiol (CBD) may help improve symptoms in people with autism, such as attention issues, sleep problems, and social interaction difficulties. In one study, most patients showed improvement in these areas after using CBD, although more research is needed to confirm these findings. ^{1 2 3 4 5}

Is cannabidiol (CBD) safe for use in humans?

Cannabidiol (CBD), also known as Epidiolex, has been studied for safety in humans, especially in treating epilepsy. Common side effects include sleepiness and diarrhea, and it can interact with other medications, like lithium, potentially causing issues. In studies with autistic patients, CBD was generally well-tolerated, with only mild side effects reported. ^{1 2 3 6 7}

How does the drug Epidiolex (Cannabidiol) differ from other treatments for autism?

Epidiolex, a highly purified form of cannabidiol (CBD), is unique because it is derived from plants and has shown promise in improving various autism symptoms, such as communication and social interaction deficits, even in non-epileptic patients. Unlike many traditional treatments, it may also allow for the reduction or withdrawal of other medications while maintaining symptom improvements. ^{1 2 3 6 8}

Research Team

Doris Trauner, MD

Principal Investigator

University of California, San Diego

Eligibility Criteria

This trial is for boys aged 7-14 with autism who exhibit severe behaviors like aggression, self-harm, and hyperactivity almost daily. They must have a confirmed diagnosis of autism based on ADOS testing and significant functional impairment due to these behaviors.

Inclusion Criteria

You often hurt yourself or others on a regular basis, almost every day.

You frequently show aggressive or self-injurious behaviors almost every day in different places like home, school, or clinic.

Boys between 7 and 14 years old.

Exclusion Criteria

Children who might travel out of the area for a significant time during the study

Children who recently participated in another investigational drug trial may be excluded

A known genetic condition such as tuberous sclerosis

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Baseline Evaluation

Baseline clinical evaluation, neuropsychological, behavioral, cognitive, and language testing, EEG, and MRI scan

1-2 weeks

1 visit (in-person)

Treatment Period 1

15 children receive CBD and 15 receive placebo for 8 weeks

8 weeks

Weekly visits (in-person)

Washout

4-week washout period with repeated behavioral and cognitive tests

4 weeks

1 visit (in-person)

Treatment Period 2

Treatments are reversed; 15 children receive CBD and 15 receive placebo for 8 weeks

8 weeks

Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

2 visits (in-person)

Treatment Details

Interventions

• Epidiolex
• Placebo Oral Solution

Trial Overview The study tests if cannabidiol (Epidiolex) can reduce severe behavioral problems in children with autism compared to a placebo. It aims to see if the drug helps with aggressive behavior, self-harm, repetitive actions, or extreme hyperactivity.

Participant Groups

Group I: cannabidiol Experimental Treatment 1 Intervention

cannabidiol arm

Group II: placebo Placebo Group 1 Intervention

placebo arm

Epidiolex is already approved in United States, European Union 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 Epidyolex for:

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

Who Is Running the Clinical Trial?

University of California, San Diego

Lead Sponsor

Trials

1,215

Recruited

1,593,000+

Wholistic Research and Education Foundation

Collaborator

Trials

1

Recruited

40+

Center for Medicinal Cannabis Research

Collaborator

Trials

19

Recruited

630+

Findings from Research

Epidiolex® (CBD) is effective for treating epilepsy in patients over 2 years old, but it can interact with other medications, as seen in a case where it contributed to lithium toxicity in a patient with Lennox-Gastaut syndrome.

After discontinuing lithium due to toxicity, the patient improved and remained seizure-free on CBD and two other antiseizure medications, highlighting the importance of monitoring drug interactions when adding CBD to treatment regimens.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Drug-Drug Interactions Between Cannabidiol and Lithium. Singh, RK., Dillon, B., Tatum, DA., et al.[2020]

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]

Pure, plant-derived cannabidiol (CBD; Epidiolex) has been shown to significantly reduce seizure frequencies in patients with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) by 17% to 23% compared to placebo, making it an effective adjunctive therapy for treatment-resistant epilepsy.

CBD is generally well tolerated, with common side effects including somnolence, diarrhea, and elevated liver enzymes, and it is the first cannabis-derived medication approved by the FDA for these conditions, providing a new treatment option for patients aged 2 years and older.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cannabidiol: A New Hope for Patients With Dravet or Lennox-Gastaut Syndromes. Chen, JW., Borgelt, LM., Blackmer, AB.[2020]

Current research indicates that cannabis and cannabidiol may offer potential therapeutic benefits for some individuals with autism spectrum disorder (ASD), particularly for associated noncore symptoms and comorbidities.

The available data suggest that these treatments are generally well tolerated, but further studies are needed to identify which patients might benefit the most and to ensure safety without adverse effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Autism Spectrum Disorder and Medical Cannabis: Review and Clinical Experience. Mostafavi, M., Gaitanis, J.[2021]

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]

A single oral dose of 750 mg of cannabidiol (CBD) showed significantly increased exposure when taken with a high-fat/calorie meal, with a 3.8-fold increase in overall exposure (AUC0-∞) and a 5.2-fold increase in maximum concentration (Cmax) compared to fasting.

CBD was well-tolerated with no severe adverse events reported, indicating its safety in healthy adults, while its absorption was also enhanced by low-fat meals, whole milk, and alcohol, albeit to a lesser extent.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase 1, randomized, pharmacokinetic trial of the effect of different meal compositions, whole milk, and alcohol on cannabidiol exposure and safety in healthy subjects. Crockett, J., Critchley, D., Tayo, B., et al.[2021]

Epidiolex, a 99% pure oral CBD extract, has been shown to significantly reduce seizure frequency in patients with medically refractory epilepsy, leading to its FDA approval.

The review highlights the safety profile of Epidiolex, noting that while it is effective, understanding its adverse effects is crucial for patient management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Epidiolex as adjunct therapy for treatment of refractory epilepsy: a comprehensive review with a focus on adverse effects. Sekar, K., Pack, A.[2020]

In a study of 47 patients with refractory epilepsy, highly purified cannabidiol (CBD) was found to be effective in reducing seizure frequency, regardless of whether it was used with clobazam (CLB) or not.

Patients taking CBD alongside CLB had a significantly higher responder rate (more than 50% reduction in seizures) at the point of best seizure control within the first year, indicating that combining these treatments may enhance efficacy.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of cannabidiol in subjects with refractory epilepsy relative to concomitant use of clobazam. Savage, TE., Sourbron, J., Bruno, PL., et al.[2021]

References

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

Drug-Drug Interactions Between Cannabidiol and Lithium. [2020]

2. 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]

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

Cannabidiol: A New Hope for Patients With Dravet or Lennox-Gastaut Syndromes. [2020]

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

Autism Spectrum Disorder and Medical Cannabis: Review and Clinical Experience. [2021]

5. England pubmed.ncbi.nlm.nih.gov

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

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

A phase 1, randomized, pharmacokinetic trial of the effect of different meal compositions, whole milk, and alcohol on cannabidiol exposure and safety in healthy subjects. [2021]

7. England pubmed.ncbi.nlm.nih.gov

Epidiolex as adjunct therapy for treatment of refractory epilepsy: a comprehensive review with a focus on adverse effects. [2020]

8. Netherlands pubmed.ncbi.nlm.nih.gov

Efficacy of cannabidiol in subjects with refractory epilepsy relative to concomitant use of clobazam. [2021]