INTERVENTIONALTERMINATEDNCT03849456

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.

Locally preserved from ClinicalTrials.govOpen ClinicalTrials.govlast source update 2022-09-02

What this record can show

Protocol only - no registry results posted

This locally preserved record separates the registered protocol from source-reported registry results. Neither is a treatment recommendation.

Registry facts

Study at a glance

Phase: PHASE2
Enrollment: 1 (ACTUAL)
Start date: 2020-01-07
Sponsor: Jazz Pharmaceuticals
Design: NA · SINGLE GROUP · TREATMENT
Locations: 3
Results record: Not present in registry snapshot

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.

Linked local records

Related local records

Local links use governed condition terms or exact source-reported intervention names. They are navigation candidates, not efficacy claims.

Conditions

No published condition report matched.

Cannabinoids and active compounds

2-[(1S,6S)-6-isopropenyl-3-methyl-1-cyclohex-2-enyl]-5-propyl-benzene-1,3-diol Cannabidivarin

Medicines

No exact medicine-name link was found.

Interventions

What was registered

DRUG

GWP42006

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

Cannabidivarin · CBDV

Eligibility

Population and criteria

Sex: ALL
Minimum age: 4 Years
Maximum age: 18 Years
Healthy volunteers: Not accepted

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.

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)

primary outcomes

primary measures

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.

secondary outcomes

secondary measures

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.