- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04517799
Trial of Cannabidiol to Treat Severe Behavior Problems in Children With Autism
A Double-Blind, Crossover Trial of Cannabidiol to Treat Severe Behavior Problems in Children With Autism
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Locations
-
-
California
-
La Jolla, California, United States, 92093
- University of California San Diego
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Boys ages 7-14 years
- Confirmed diagnosis of autism based on ADOS testing
- Autism severity assessed as severe with substantial behavioral problems
Severity of symptoms will be based on a number of criteria:
- Aggressive and/or self-injurious (SIBs) behaviors occur almost daily (more than 6 times per week) in any situation (home, school, clinic, etc.).
- Frequent (daily), persistent (lasting at least 5-10 minutes and repeated through the day) stereotypies (repetitive behaviors such as hand flapping, running in circles, jumping repeatedly, waving fingers in front of eyes)
- Pervasive hyperactivity (child is so physically active that he cannot sit for meals or school work, is moving all the time, jumping off furniture, climbing onto furniture, etc.)
- One of more of the above activities is deemed to contribute significantly to child's inability to function by parental report and with clinician agreement based on history and/or direct observation
Exclusion Criteria:
- the presence of epilepsy
- a known genetic condition such as tuberous sclerosis
- other significant health issues such as cardiac disease, presence of known congenital brain malformation, or a history of central nervous system infection.
- children who are on anticonvulsant medications such as clobazam or valproic acid will also be excluded because of potential drug-drug interactions. At the time of screening, each child's medication list will be checked for drugs that are known to cause interactions with cannabidiol.
- children with an allergy to any components of the study drug
- children who are taking CBD from another source, unless parents are willing to stop the treatment for at least 4 weeks prior to entering the study. CBD and other cannabinoid blood levels will be performed at baseline and if CBD is detected in the blood, the child will be not be included in the study.
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: placebo
placebo arm
|
GW has developed a placebo that is identical in color and taste, and will be administered in the same volume as the CBD, so that parents and investigators should not be able to detect differences between the 2 treatments. Dosing is dependent upon each subject's weight. The dosage will be titrated as follows: Week 1: 5 mg/kg/day, divided into 2 doses Week 2: 10 mg/kg/day, divided into 2 doses Weeks 3-8: 20 mg/kg/day, divided into 2 doses If a child's weight changes by greater than 2 kg during the study, the dose will be adjusted to reflect the weight change. |
Experimental: cannabidiol
cannabidiol arm
|
While in the intervention arm subjects will receive EPIDIOLEX, an oral purified CBD solution developed by GW Pharmaceuticals. The formulation is a 100 mg/mL solution, which allows for easy dose titration and administration even to young children. EPIDIOLEX is naturally-derived CBD, and has been found to have a purity of over 98%. The solution includes a sweetener to make it palatable. Dosing is dependent upon each subject's weight. The dosage will be titrated over time as follows: Week 1: 5 mg/kg/day, divided into 2 doses Week 2: 10 mg/kg/day, divided into 2 doses Weeks 3-8: 20 mg/kg/day, divided into 2 doses If a child's weight changes by greater than 2 kg during the study, the dose will be adjusted to reflect the weight change.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Total Score on Repetitive Behavior Scale-Revised (RBS-R)
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
This scale aims to capture the frequency and severity of the repetitive and restricted behaviors that are a common feature in individuals with autism spectrum disorders.
The RBS-R has been validated and has been shown to have high internal consistency and inter-rater reliability when used in outpatient settings.
There are 5 subscales: Stereotypic Behavior, Self-Injurious Behavior, Compulsive Behavior, Ritualistic/Sameness Behavior, and Restricted Interests.
This scale will be administered to the parent or caregiver, who will report on the child's recent behaviors.
Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Total Score on Child Behavior Checklist (CBCL)
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
The CBCL is a widely used test that assesses problem behaviors in children by posing questions to the parent or caregiver.
It poses questions about a wide range of potential negative behaviors including aggression, rule-breaking, and social problems.
The school-aged scale, which will be used in this study, consists of 118 questions.
The CBCL has 8 syndrome scales, including Aggressive Behavior, Anxiety/Depressed, Attention Problems, Rule-breaking, Somatic Complaints, Social Problems, Thought Problems, and Withdrawn/Depressed. Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Autism Diagnostic Observation Scale- 2 (ADOS-2)
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
This a widely used scale that diagnoses autism spectrum disorders across individuals with a broad range of ages, developmental levels, and language abilities.
It is a semi-structured assessment and consists of behavioral observation and coding of relevant behaviors.
The ADOS-2 evaluates communication, social interaction, and imaginative play.
The child is asked to complete various activities and then rated on their performance by the tester.
Time of administration is 40-60 minutes.
Range of scores is 1-10.
Changes in score from baseline to end of treatment will be estimated - one observed during the period of treatment with the Placebo and another during the CBD treatment - with negative value representing improvement in negative behaviors and positive value representing worsening of those characteristics.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Total Score on Aberrant Behavior Checklist (ABC)
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
This checklist relies upon parent/caregiver report to assess problematic behaviors.
These behaviors are divided into 5 subscales: irritability, social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Total Score on Self Harm Inventory
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
This is a one-page, 22-item questionnaire that poses yes/no questions about the subject's history of self-harm.
Total score is the summary of "yes" responses.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Total Score on Social Responsiveness Scale
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Formerly known as the Social Reciprocity Scale, this 65 item questionnaire takes a quantitative approach to measuring autistic symptomatology.
It obtains "first-hand" ratings from individuals (in this case, parents) who have observed the child in naturalistic social settings.
The SRS poses questions about a child's ability to engage in emotionally appropriate reciprocal social interactions, It generates a singular severity score for autistic social impairment (higher score equates with greater severity), use of which has been validated by extensive factor and cluster analyses among clinical and nonclinical populations.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Total Score on Peabody Picture Vocabulary test (PPVT-4)
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
The PPVT-4 is a measure of receptive vocabulary for Standard American English.
For this study we will use standard scores.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Total Score on Expressive One Word Picture Vocabulary test, Fourth Edition (EOWPVT-4)
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
This test assesses the individual's ability to name a variety of objects, actions, and concepts.
Participants in this study are expected to have limited verbal abilities.
This test will evaluate the degree of impairment at baseline and then following treatment.
Administration takes less than twenty minutes.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Total Score on Children's Sleep Habits Questionnaire (CSHQ)
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
This is a retrospective, 45-item parent questionnaire that has been used in a number of studies to examine sleep behavior in young children.
Thirty-two items on the CSHQ are grouped into 8 subscales relating to a number of key sleep domains: (1) bedtime resistance (6 items); (2) sleep-onset delay (1 item); (3) sleep duration (3 items); (4) sleep anxiety (4 items); (5) night wakings (3 items); (6) parasomnias (7 items); (7) sleep-disordered breathing (2 items); and (8) daytime sleepiness (8 items).
Parents are asked to recall sleep behaviors occurring during a typical recent week.
A higher score is indicative of more disturbed sleep.
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Magnetic Resonance Spectroscopy
Time Frame: Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Analyses will include levels of GABA and other compounds that can be identified in MR spectra; changes in those compounds with treatment; and connectivity of different brain regions before and after treatment.
MRS will examine the peaks of various neurotransmitters (glutamate, glutamine, markers of neuronal integrity (NAA) and markers of metabolic function (lactate, alanine).
|
Start to finish of period 1 (8 weeks) and start to finish of period 2 (8 weeks)
|
Collaborators and Investigators
Collaborators
Investigators
- Principal Investigator: Doris Trauner, MD, University of California, San Diego
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 181455
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Autism Spectrum Disorder
-
Stanford UniversityCalifornia Department of Developmental ServicesRecruitingAutism Spectrum Disorder | Autistic Disorder | Autism | Autism Spectrum Disorders | Autistic Disorders Spectrum | Autistic Spectrum Disorder | Autistic Spectrum DisordersUnited States
-
Hoffmann-La RocheActive, not recruitingAutism Spectrum Disorder (ASD)United States, Canada, Italy, Spain
-
Axial Therapeutics, Inc.Active, not recruitingAutism Spectrum Disorder (ASD)United States, Australia, New Zealand
-
Technion, Israel Institute of TechnologyCompleted
-
Stanford UniversityNational Institute on Deafness and Other Communication Disorders (NIDCD)CompletedAutism | Autism Spectrum Disorder (ASD)United States
-
Corporacion Parc TauliUnknown
-
Institut de Recherches Internationales ServierADIR, a Servier Group companyTerminatedAutism Spectrum Disorder (ASD)Spain, United States, Hungary, Poland, Australia, United Kingdom, Brazil, Czechia, France, Italy, Portugal, Slovakia
-
Florida Gulf Coast UniversityCompletedAutism Spectrum Disorder High-FunctioningUnited States
-
Hospital Universitario Dr. Jose E. GonzalezUnknownAutism | Autism SpectrumMexico
-
National Taiwan University HospitalCompletedAutism Spectrum Disorder High-FunctioningTaiwan
Clinical Trials on Epidiolex
-
Augusta UniversityState of GeorgiaAvailable
-
Jazz PharmaceuticalsRecruitingTuberous Sclerosis Complex Associated Neuropsychiatric DiseaseUnited States, United Kingdom, Poland
-
Massachusetts Institute of TechnologyMassachusetts General HospitalNot yet recruitingPhobia, SocialUnited States
-
Mayo ClinicCompletedUrinary StoneUnited States
-
University of South FloridaJazz PharmaceuticalsCompletedStudy the Efficacy of Epidiolex for Typical Absence SeizuresUnited States
-
Mclean HospitalCompletedCannabis Use DisorderUnited States
-
Eric Marsh, MDUniversity of Pittsburgh; Geisinger ClinicNo longer availableDrug Resistant Epilepsy
-
Kevin BoehnkeMichigan, State of, Licensing and Regulatory AffairsRecruiting
-
University of California, San DiegoRecruiting
-
University of ChicagoRecruitingObsessive-Compulsive Disorder | Tourette Syndrome | Trichotillomania (Hair-Pulling Disorder) | Hoarding DisorderUnited States