Safety and Tolerability of Cannabidivarin (CBDV) in Children and Young Adults With Autism Spectrum Disorder

August 31, 2022 updated by: Jazz Pharmaceuticals

Safety and Tolerability of GWP42006 in Children and Young Adults With Autism Spectrum Disorder

To determine the safety and tolerability of GWP42006 (cannabidivarin, CBDV) in children and young adults with autism spectrum disorder (ASD) and to examine the effect of GWP42006 on communication, social interactions, sleep, behavior, and cognition profiles.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This is a 52-week, open-label trial to evaluate the safety and tolerability of GWP42006. Participants who satisfy all eligibility criteria will start GWP42006 at a dose of 2.5 milligrams per kilogram per day (mg/kg/day) and titrate to a target dose of 10 mg/kg/day or 800 mg/day, whichever is smaller, during the first 4 weeks of treatment. If there is intolerance during titration, the participant may be maintained on a dose below 10 mg/kg/day. The maximum dose participants aged 6 years or older can receive will be 20 mg/kg/day or 1600 mg/day, whichever is smaller. Following the final treatment dose, participants will taper GWP42006 10% per day. The investigator will withdraw participants who fail to demonstrate any perceived benefit and may withdraw participants for whom tolerability is poor.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
1

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Clinical Trial Site
    • Massachusetts
      • Lexington, Massachusetts, United States, 02421
        • Clinical Trial Site
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Clinical Trial Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 months to 14 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Key Inclusion Criteria:

  • Diagnosis of Autism Spectrum Disorder (ASD) as defined by the Autism Diagnostic Observation Schedule, 2nd Edition and The Diagnostic and Statistical Manual of Mental Disorders, 5th Edition
  • Intelligence quotient (IQ) of 40-120 (inclusive)
  • Participant and their caregiver are English speaking.
  • In the opinion of the investigator, the participant presents with ASD symptoms that warrant a therapeutic trial with GWP42006.

Key Exclusion Criteria:

  • Known single gene neurogenetic disorder with high rates of epilepsy/autism (e.g., fragile X, tuberous sclerosis complex), structural brain lesion (prior stroke or hemispheric brain malformations), or history of any other epileptic encephalopathy, including infantile spasms, before the diagnosis of ASD
  • More than 2 epileptic seizures per month within the 6 months prior to screening
  • Initiation of a behavioral therapy program, new psychotropic medication, or therapeutic diet within the 2 months prior to screening, or plan to change or start any of the above during the trial
  • Presence of a significant untreated medical problem (obstructive sleep apnoea, restless legs syndrome, gastroesophageal reflux disease, etc.) which may have significant impact on sleep study measures
  • Behavioral management issues (e.g., self-injury, aggression) severe enough to be of safety concerns (to participant and/or staff)
  • Clinically significant electrocardiogram abnormality or postural drop in systolic blood pressure at screening
  • Any known or suspected hypersensitivity to cannabinoids or any of the excipients of GWP42006, such as sesame oil
  • Known history of psychiatric disorder (defined as schizophrenia, bipolar disorder, or other psychiatric disease with a known history of hallucinations or delusions)
  • History of any inborn errors of metabolism
  • Significantly impaired hepatic function at screening
  • Received an investigational product within the 3 months prior to screening
  • Participant has been taking felbamate for less than 1 year prior to screening
  • History of substance use disorders or positive drug of abuse dipstick test at screening (unless the positive result is due to a known concomitant medication)
  • Currently using or has used recreational or medicinal cannabis or cannabinoid-based medications within the 3 months prior to screening and is unwilling to abstain for the duration of the trial
  • Any history of suicidal behavior or any suicidal ideation within the month prior to or at screening

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: GWP42006
Oral solution taken twice daily with food for 52 weeks.
Drug: GWP42006
Oral solution containing cannabidivarin 50 milligrams per milliliter (mg/mL) in sesame oil with anhydrous ethanol, sucralose, strawberry flavoring, and β-carotene.
Other Names:
  • Cannabidivarin
  • CBDV

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)
Time Frame: Day 1 to Day 403
A TEAE was defined as an adverse event (AE) with an onset date on or after the first dose of GWP42006. If an AE had a partial onset date and it was unclear from the partial date (or the stop date) whether the AE started prior to or following the first dose of GWP42006, then the AE was considered a TEAE. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through to the Safety Follow-up will be presented.
Day 1 to Day 403

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change from Baseline in Children's Communication Checklist-2 (CCC-2)
Time Frame: Baseline to End of Treatment (Day 365) or Early Termination (ET)
The CCC-2 is a caregiver-completed measure designed to assess the communication skills of children 4 to 16 years of age. An increase in score indicates an improvement.
Baseline to End of Treatment (Day 365) or Early Termination (ET)
Change from Baseline in Social Responsiveness Scale-2 (SRS-2)
Time Frame: Baseline to End of Treatment (Day 365) or ET
The SRS-2 assesses social impairment associated with Autism Spectrum Disorder (ASD). Total scores between 58 and 72 indicate a mild deficiency, between 73 and 97 indicate a moderate deficiency, and 98 or above indicate a severe deficiency. A decrease in total score indicates an improvement.
Baseline to End of Treatment (Day 365) or ET
Change from Baseline in Vineland Adaptive Behavior Scales, 3rd Edition (Vineland-3)
Time Frame: Baseline to End of Treatment (Day 365) or ET
The Vineland-3 measures the personal and social skills of individuals from birth through adulthood. Score ranges are as follows: 70 to 80, borderline adaptive functioning; 51 to 70, mildly deficient adaptive functioning; 35 to 50, moderately deficient adaptive behavior; 20 to 35, severely deficient adaptive behavior; less than 20, markedly or profoundly deficient adaptive behavior.
Baseline to End of Treatment (Day 365) or ET
Change from Baseline in National Institutes of Health (NIH) Toolbox Cognition Battery
Time Frame: Baseline to End of Treatment (Day 365) or ET
The NIH Toolbox Cognition Battery has computerized instruments that measure several ability subdomains important for cognitive health.
Baseline to End of Treatment (Day 365) or ET
Change from Baseline in Repetitive Behavioral Scale - Revised (RBS-R)
Time Frame: Baseline to End of Treatment (Day 365) or ET
The RBS-R is used to measure the breadth of repetitive behavior in children, adolescents, and adults with ASD. Total scores range from 0 to 129. A decrease in total score indicates an improvement.
Baseline to End of Treatment (Day 365) or ET
Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ)
Time Frame: Baseline to End of Treatment (Day 365) or ET
The CSHQ is used to examine sleep behavior in young children. Responses are given on a 5-point scale (1 = always; 5 = never). A higher score reflects more sleep problems.
Baseline to End of Treatment (Day 365) or ET
Change from Baseline in Aberrant Behavior Checklist (ABC)
Time Frame: Baseline to End of Treatment (Day 365) or ET
The ABC assesses the presence and severity of various problem behaviors commonly observed in individuals diagnosed with intellectual and developmental disabilities. Total scores range from 0 to 174. A decrease in total score indicates an improvement.
Baseline to End of Treatment (Day 365) or ET
Clinical Global Impressions-Improvement (CGI-Improvement)
Time Frame: Baseline to End of Treatment (Day 365) or ET
The CGI-Improvement evaluates the change from the initiation of treatment in the severity of psychopathology using a 7-point scale ranging from 1 (very much improved) to 7 (very much worse). Lower scores represent an improvement.
Baseline to End of Treatment (Day 365) or ET

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jazz Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 7, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
May 26, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 26, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 20, 2019

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 20, 2019

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 21, 2019

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 2, 2022

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 31, 2022

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2022

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GWND18089

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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.

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