AzTrials
AzTrials

Autism Spectrum DisorderPhase 2NCT03849456

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

A Phase 2 study testing GWP42006 in people with Autism Spectrum Disorder, run by Jazz Pharmaceuticals at 3 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

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. Read more about this study

Who is it for?
Ages 4 to 18 years
Conditions the study is for
Autism Spectrum Disorder
What is being tested?
GWP42006
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
1 participants
Where
3 sitesUnited States
Study dates
7 Jan 2020to 26 May 2020
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 4 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

  • 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
Show all 15Show fewer
  • 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

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Clinical Trial Site

    Columbus, Ohio, 43205

  • Clinical Trial Site

    Lexington, Massachusetts, 02421

  • Clinical Trial Site

    Louisville, Kentucky, 40202

Other trials in United States

About this study

Official title: 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.

Read the 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.

Being tested
GWP42006
Keywords
Cannabidivarin, CBDV, GWP42006
Medical subject headings
Autism Spectrum Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GWP42006Drug

    Also called Cannabidivarin, CBDV

    Oral solution containing cannabidivarin 50 milligrams per milliliter (mg/mL) in sesame oil with anhydrous ethanol, sucralose, strawberry flavoring, and β-carotene.

Study groups in NCT03849456
Study groupRoleWhat they receive
GWP42006ExperimentalOral solution taken twice daily with food for 52 weeks.
Receives: Drug: GWP42006

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs)

    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.

    Measured: Day 1 to Day 403

Secondary outcomes (8)

  1. Change from Baseline in Children's Communication Checklist-2 (CCC-2)

    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.

    Measured: Baseline to End of Treatment (Day 365) or Early Termination (ET)

  2. Change from Baseline in Social Responsiveness Scale-2 (SRS-2)

    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.

    Measured: Baseline to End of Treatment (Day 365) or ET

  3. Change from Baseline in Vineland Adaptive Behavior Scales, 3rd Edition (Vineland-3)

    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.

    Measured: Baseline to End of Treatment (Day 365) or ET

  4. Change from Baseline in National Institutes of Health (NIH) Toolbox Cognition Battery

    The NIH Toolbox Cognition Battery has computerized instruments that measure several ability subdomains important for cognitive health.

    Measured: Baseline to End of Treatment (Day 365) or ET

  5. Change from Baseline in Repetitive Behavioral Scale - Revised (RBS-R)

    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.

    Measured: Baseline to End of Treatment (Day 365) or ET

  6. Change from Baseline in Children's Sleep Habits Questionnaire (CSHQ)

    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.

    Measured: Baseline to End of Treatment (Day 365) or ET

  7. Change from Baseline in Aberrant Behavior Checklist (ABC)

    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.

    Measured: Baseline to End of Treatment (Day 365) or ET

  8. Clinical Global Impressions-Improvement (CGI-Improvement)

    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.

    Measured: Baseline to End of Treatment (Day 365) or ET

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Feb 2019
  2. Study start7 Jan 2020
  3. Primary completion26 May 2020
  4. Study completion26 May 2020

Why it stopped: Study terminated due to enrollment challenges during COVID-19 pandemic.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
1 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03849456
Sponsor's protocol ID
GWND18089
Status last verified
Aug 2022
First submitted
20 Feb 2019
Last updated
2 Sep 2022
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing GWP42006 in people with Autism Spectrum Disorder, run by Jazz Pharmaceuticals at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 2 Sep 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Study terminated due to enrollment challenges during COVID-19 pandemic.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants Who Experienced Severe Treatment-Emergent Adverse Events (TEAEs) (time frame: Day 1 to Day 403). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03849456.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Sep 2022.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in