Cannabidivarin for treating irritability in children with autism

Cannabidivarin (CBDV) vs. Placebo in Children With Autism Spectrum Disorder (ASD)

This study is testing if cannabidivarin can help reduce irritability in children with autism without the side effects of current medications.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 5 Years to 18 Years
• Sex: All
• Sponsor: Montefiore Medical Center (Academic / other)
• Locations: 2 sites (New York, New York and 1 other locations)
• Trial ID: NCT03202303 on ClinicalTrials.gov

What this trial studies

This trial evaluates the safety and efficacy of cannabidivarin (CBDV), a nonpsychoactive cannabinoid, in children diagnosed with Autism Spectrum Disorder (ASD). The study aims to address the unmet need for effective treatments that do not carry the adverse metabolic side effects associated with current FDA-approved medications. Participants will be pediatric outpatients aged 5 to 18 with confirmed ASD and significant irritability, as measured by specific behavioral scales. The trial will compare the effects of CBDV against a matched placebo over a defined period.

Who should consider this trial

Good fit: Ideal candidates are children aged 5 to 18 with a confirmed diagnosis of ASD and significant irritability.

Not a fit: Patients who do not exhibit significant irritability or those with contraindications to cannabinoid treatments may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a safer alternative for managing irritability in children with ASD without the adverse effects of current medications.

How similar studies have performed: While there is limited research on CBDV specifically, other cannabinoid-based approaches have shown promise in treating symptoms of ASD, indicating potential for success.

Eligibility criteria

Inclusion Criteria

1. Male or Female pediatric outpatients aged between and including ages 5 to 18. Diagnosis of Autism Spectrum Disorder (ASD) confirmed by the ADOS-2 and DSM-5 criteria.\*During special circumstances (e.g. COVID-19 pandemic) where the ADOS-2 cannot be performed due to site restrictions (e.g. mandatory use of face masks), eligibility can be confirmed using the Autism Diagnostic Interview, Revised (ADI-R)
2. Aberrant Behavior Checklist (ABC) - Irritability Subscale (ABC-I) score of 18 or greater at screening visit.
3. Social Responsiveness Scale (SRS) score of 66T or higher at screening visit.
4. Clinical Global Impression Scale - Severity (CGI-S) score of 4 or higher at screening.
5. Stable pharmacologic, educational, behavioral and/or dietary interventions for 4 weeks prior to randomization and for the duration of the study.
6. Physical exam and laboratory results that are within normal range for individuals with ASD.
7. Presence of a parent/caregiver/guardian that is able to consent for their participation and complete assessments regarding the child's development and behavior throughout the study. Child Assent will be obtained if the subject is 7 years of age or older and has the mental capacity to understand and sign a written assent form and/or give verbal assent.

Exclusion Criteria

1. Exposure to any investigational agent in the 30 days prior to randomization.
2. Prior chronic treatment with CBD, CBDV or an endocannabinoid treatment.
3. Positive testing for THC or other drugs of abuse via urine testing at the screening visit or baseline visits upon repeat confirmation testing.
4. Recent history of drug abuse including marijuana/cannabis use in the past 3 months.
5. Diagnosis of a known genetic disorder (ie. Prader-Willi Syndrome, Angelman Syndrome etc.).
6. A primary psychiatric diagnosis other than ASD, including bipolar disorder, psychosis, schizophrenia, Post-Traumatic Stress Disorder (PTSD) or Major Depressive Disorder (MDD). These patients will be excluded due to potential confounding results.
7. A medical condition that severely impacts the subject's ability to participate in the study, interferes with the conduct of the study, confounds interpretation of study results or endangers the subject's well-being.
8. A known diagnosis of Rett Syndrome or Childhood Disintegrative Disorder, or marked sensory impairment such as deafness or blindness.
9. Subjects who have had changes in allied health therapies, behavioral or educational interventions within four weeks prior to randomization other than those associated with school holidays.
10. Subjects who have had changes in medications or medication doses within four weeks of randomization. Renal, pancreatic, or hematologic dysfunction as evidenced by values above upper limits of normal for BUN/creatinine, or values twice the upper limit of normal for serum lipase and amylase, platelets \<80,000 /mcL, or WBC\<3.0 103 /mcL
11. Liver dysfunction manifested by \> 2 X UNL values of AST or ALT
12. ECG abnormality at baseline screening or clinically significant postural drop in systolic blood pressure at screening. If the initial screening ECG show a QTcB of greater than 460 msec, then 2 additional ECGs will be conducted in the same sitting, 5 minutes apart. If not recognized at screening, then a full triplicate repeat showing an average QTcB of 460 msec or less to meet all inclusion/exclusion criteria. Female subjects who are pregnant will be excluded from the study. If a female subject is able to become pregnant, she will be given a serum pregnancy test before entry into the study. Female subjects will be informed not to become pregnant while taking CBDV. Female subjects must tell the investigator and consult an obstetrician or maternal-fetal specialist if they become pregnant during the study.
13. Known allergy to sesame oil

Where this trial is running

New York, New York and 1 other locations

• New York University (NYU) Langone — New York, New York, United States (Completed)
• Montefiore Medical Center — The Bronx, New York, United States (Recruiting)

Study contacts

• Principal investigator: Eric Hollander, MD — Montefiore Medical Center/Albert Einstein College of Medicine
• Study coordinator: Casara Ferretti, MS, MA

Conditions Autism Spectrum Disorder; Autism; Irritability; Cannabinoids