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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04745026




Registration number
NCT04745026
Ethics application status
Date submitted
4/02/2021
Date registered
9/02/2021

Titles & IDs
Public title
Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder
Scientific title
An Exploratory, Phase 2, Randomized, Double-blind, Placebo-controlled Trial to Investigate the Safety and Efficacy of Cannabidiol Oral Solution (GWP42003-P; CBD-OS) in Children and Adolescents With Autism Spectrum Disorder
Secondary ID [1] 0 0
2020-002819-21
Secondary ID [2] 0 0
GWND19189
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autism Spectrum Disorder 0 0
Condition category
Condition code
Mental Health 0 0 0 0
Autistic spectrum disorders
Mental Health 0 0 0 0
Other mental health disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GWP42003-P
Treatment: Drugs - Placebo

Experimental: GWP42003-P 10 mg/kg/day - Participants will be stratified based on their age (6 to 11 years old, 12 to 17 years old), use of antipsychotics (on versus off), and region (North America versus Rest of the World) and will be randomized to receive 5 milligrams per kilogram per day (mg/kg/day) GWP42003-P for 1 week and then 10 mg/kg/day GWP42003-P for 11 weeks.

Placebo comparator: Placebo - Participants will be stratified based on their age (6 to 11 years old, 12 to 17 years old), use of antipsychotics (on versus off), and region (North America versus Rest of the World) and will be randomized to receive matching placebo for 12 weeks.


Treatment: Drugs: GWP42003-P
GWP42003-P oral solution (100 milligrams per milliliter \[mg/mL\] cannabidiol \[CBD\] in sesame oil with anhydrous ethanol, ethanol \[10% v/v\] sweetener \[sucralose\], and strawberry flavoring), administered twice a day (morning and evening)

Treatment: Drugs: Placebo
Oral placebo to match GWP42003-P oral solution containing sesame oil with anhydrous ethanol, sweetener (sucralose), strawberry flavoring, and beta carotene, administered twice a day (morning and evening)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Aberrant Behavior Checklist (ABC) Subscale Total Scores
Assessment method [1] 0 0
The caregiver-assessed ABC was designed to assess the presence and severity of various problem behaviors commonly observed in individuals diagnosed with intellectual and developmental disability. The checklist contains 5 subscales: Irritability (15 items); Social Withdrawal (16 items); Stereotypic Behavior (7 items); Hyperactivity/Noncompliance (16 items); and Inappropriate Speech (4 items). Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). The total score of all items for each subscale range from 0-45 (irritability), 0-48 (social withdrawal and hyperactivity/noncompliance), 0-21 (stereotypic behavior), and 0-12 (inappropriate speech) where higher scores indicate worse clinical outcome. The change from baseline to Week 4, Week 8, and Week 12 is reported with lower scores indicating better clinical outcome.
Timepoint [1] 0 0
Baseline up to Week 12
Primary outcome [2] 0 0
Change From Baseline in Vineland Adaptive Behavior Scales-3 (VABS-3) Scores
Assessment method [2] 0 0
The VABS-3 scales assess what a person does, rather than what he or she can do. The Vineland-3 assesses adaptive behavior in 3 domains: Communication, Daily Living Skills, and Socialization. Each domain is comprised of 3 subdomains: receptive expression and written (communication); personal, domestic and community (daily living skills); Interpersonal relationships, play and leisure and copying skills (socialization). The adaptive behavior composite score is calculated as arithmetic mean of all 3 domain scores. The total score range is 20 to 140, where low scores indicate low (worst) clinical outcome and high scores indicate high (best) clinical outcome. The change from baseline in VABS-3 is being reported with positive values indicating a positive improvement in adaptive behavior.
Timepoint [2] 0 0
Baseline up to Week 12
Primary outcome [3] 0 0
Number of Patients Per Clinical Global Impression Improvement (CGI-I) Category
Assessment method [3] 0 0
The CGI-I is a 7-point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention. The clinician is asked: Compared to the patient's condition at admission to the project, how much has the patient changed? This is rated as: 1 = very much improved; 2 = much improved; 3 = minimally improved; 4 = no change; 5 = minimally worse; 6 = much worse; or 7 = very much worse. Higher scores indicate worse clinical outcome. The number of patients in each CGI-I category is reported.
Timepoint [3] 0 0
Day 85
Primary outcome [4] 0 0
Change From Baseline in Clinical Global Impression Severity (CGI-S) Scores
Assessment method [4] 0 0
The CGI-S is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's experience with patients who have the same diagnosis. The clinician is asked: Considering your total clinical experience with this particular population, how ill is the patient at this time? This is rated as: 1 = normal, not at all ill; 2 = borderline ill; 3 = mildly ill; 4 = moderately ill; 5 = markedly ill; 6 = severely ill; or 7 = extremely ill. Higher scores indicate worse outcome. The change from baseline in CGI-S scores is reported and lower mean scores indicate better outcome.
Timepoint [4] 0 0
Baseline up to Week 12
Secondary outcome [1] 0 0
Number of Participants Reporting Treatment-emergent Adverse Events
Assessment method [1] 0 0
A TEAE is one that started, or worsened in severity or seriousness, following the first dose of IMP. AEs were coded according to the Medical Dictionary for Regulatory Activities v24.0 dictionary.
Timepoint [1] 0 0
Baseline up to Week 12
Secondary outcome [2] 0 0
Mean Change From Baseline in Hematology Clinical Laboratory Levels
Assessment method [2] 0 0
Timepoint [2] 0 0
Baseline up to Week 9 (end of taper/withdrawal)
Secondary outcome [3] 0 0
Mean Percentage Change From Baseline in Hematology Clinical Laboratory Levels
Assessment method [3] 0 0
Timepoint [3] 0 0
Baseline up to Week 9 (end of taper/withdrawal)
Secondary outcome [4] 0 0
Mean Change From Baseline in Hemoglobin Levels
Assessment method [4] 0 0
Timepoint [4] 0 0
Week 9 (end of taper/withdrawal)
Secondary outcome [5] 0 0
Mean Change From Baseline in Erythrocyte Mean Corpuscular Hemoglobin Levels
Assessment method [5] 0 0
Timepoint [5] 0 0
Week 9 (end of taper/withdrawal)
Secondary outcome [6] 0 0
Mean Change From Baseline in Erythrocyte Mean Corpuscular Volume Levels
Assessment method [6] 0 0
Timepoint [6] 0 0
Week 9 (end of taper/withdrawal)
Secondary outcome [7] 0 0
Number of Patients With Clinically Significant Vital Sign Values
Assessment method [7] 0 0
Timepoint [7] 0 0
Post-baseline up to 12 weeks
Secondary outcome [8] 0 0
Number of Patients With Clinically Significant Physical Examination Procedure Findings
Assessment method [8] 0 0
Number of patients with abnormal physical exam findings are reported.
Timepoint [8] 0 0
Baseline up to Day 85 post-baseline
Secondary outcome [9] 0 0
Number of Patients With Clinically Significant 12-lead Electrocardiogram Findings
Assessment method [9] 0 0
Timepoint [9] 0 0
Baseline up to Day 85 post-baseline
Secondary outcome [10] 0 0
Number of Participants Reporting Suicidal Ideation or Behavior Using the Columbia-Suicide Severity Rating Scale (C-SSRS)
Assessment method [10] 0 0
C-SSRS rating scale results since last visit is reported. The presence/absence of any suicidal ideation or behavior is scored based on yes/no responses. The overall number of participants reporting suicidal ideation or behavior is being reported.
Timepoint [10] 0 0
Baseline up to Day 92

Eligibility
Key inclusion criteria
* Participant weight is at least 12 kilograms (kg).
* Participants (if possessing adequate understanding, in the investigator's opinion) and their parent(s)/legal representative are willing and able to give informed assent and consent for participation in the trial.
* Participant and their caregiver are willing and able (in the investigator's opinion) to comply with all trial requirements.
* Participant has a diagnosis of Autism Spectrum Disorder (ASD) as per Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria for ASD, confirmed by Autism Diagnostic Observational Schedule (ADOS-2) criteria (conducted within 2 years at the trial site or at screening by a qualified assessor). Note: 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) and an ADOS- 2 conducted within 2 years at the trial site by a qualified assessor is not available, eligibility can be confirmed using: 1) an ADOS-2 performed within 2 years by a qualified assessor (external to the site); 2) if 1) is not available, eligibility may be confirmed using the Autism Diagnostic Interview, Revised (ADI-R) at screening.
* Clinical Global Impressions - Improvement Scale (CGI-S) = 4 (moderately ill) at screening and randomization.
* Intelligence quotient (IQ) = 70 at screening, or measured within 1 year of screening, using Wechsler Abbreviated Scale of Intelligence Scale Second Edition (WASI-II).
* All medications or interventions (including psychosocial interventions, dietary supplements, probiotics, speech therapy, etc.) for ASD related symptoms must have been stable for 4 weeks prior to screening and randomization, and the patient/caregiver should be willing to maintain a stable regimen throughout the trial.
* Participants must have the ability to swallow the investigational medicinal product (IMP), provided as a liquid solution.
* Participant and/or parent(s)/legal representative is willing to allow the responsible authorities to be notified of participation in the trial, if mandated by local law.
* Participant and/or parent(s)/legal representative is/are willing to allow the participant's primary care practitioner (if they have one) and consultant (if they have one) to be notified of participation in the trial if the primary care practitioner/consultant is different from the investigator.
Minimum age
6 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Current diagnosis of bipolar disorder, psychosis, schizophrenia, schizoaffective disorder, or major depression (participants with depression in remission may be included)
* Has a diagnosis other than ASD that dominates the clinical presentation (e.g., Attention Deficit Hyperactivity Disorder [ADHD])
* Has a progressive neurological condition
* Seizures in the past 24 weeks
* Changes in anticonvulsive therapy within the last 12 weeks
* Currently taking more than 2 anti-epileptic drugs (AEDs)
* Taking sirolimus, everolimus, temsirolimus, or tacrolimus
* Taking clobazam
* Taking omeprazole, lansoprazole, tolbutamide, or warfarin
* Taking repaglinide, pioglitazone, rosiglitazone, montelukast, bupropion, or efavirenz
* Currently using or has used recreational or medicinal cannabis, cannabinoid-based medications (including Sativex®, or Epidiolex®) within the 12 weeks prior to screening and is unwilling to abstain for the duration of the trial
* Participant has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP, such as sesame oil.
* Participant has moderately impaired hepatic function at screening, defined as serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 × upper limit of normal (ULN) or total bilirubin (TBL) > 2 × ULN. This criterion can only be confirmed once the laboratory results are available; participants enrolled into the trial who are later found to meet this criterion must be screen-failed.
* Participant is male and fertile (i.e., after puberty unless permanently sterile by bilateral orchidectomy) unless willing to ensure that they use male contraception (condom) or remain sexually abstinent during the trial and for 12 weeks thereafter.
* Participant is female and of childbearing potential (i.e., following menarche and until becoming postmenopausal for = 12 consecutive months unless permanently sterile by hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) unless willing to ensure that they use a highly effective method of birth control (e.g., hormonal contraception, intrauterine device/hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence) during the trial and for 12 weeks thereafter.
* Female participant who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the trial or within 12 weeks thereafter.
* Participant has received an IMP within the 12 weeks prior to the screening visit.
* Participant had brain surgery or traumatic brain injury within 1 year of screening.
* Participant has any other significant disease or disorder which, in the opinion of the investigator, may either put the participant, other participants, or site staff at risk because of participation in the trial, may influence the result of the trial, or may affect the participant's ability to take part in the trial.
* Any abnormalities identified following a physical examination of the participant that, in the opinion of the investigator, would jeopardize the safety of the participant if they took part in the trial
* Any history of suicidal behavior (lifelong) or any suicidal ideation of type 4 or 5 on the Columbia-Suicide Severity Rating Scale (C-SSRS) in the last 4 weeks or at screening or randomization
* Participant has donated blood during the past 12 weeks and is unwilling to abstain from donation of blood during the trial.
* Participant has any known or suspected history of alcohol or substance abuse or positive drugs of abuse test at screening (not justified by a known concurrent medication).
* Participant has previously been randomized into this trial.
* Participant has plans to travel outside their country of residence during the trial, unless the participant has confirmation that the IMP is permitted in the destination country/state

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/05/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
21/12/2023
Sample size
Target
Accrual to date
Final
103
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Monash Medical Centre - Clayton
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
03168 - Clayton
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Kentucky
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Washington
Country [10] 0 0
Canada
State/province [10] 0 0
Ontario
Country [11] 0 0
Germany
State/province [11] 0 0
Freiburg
Country [12] 0 0
Germany
State/province [12] 0 0
Mannheim
Country [13] 0 0
Spain
State/province [13] 0 0
Barcelona
Country [14] 0 0
Spain
State/province [14] 0 0
Sabadell
Country [15] 0 0
United Kingdom
State/province [15] 0 0
Glasgow
Country [16] 0 0
United Kingdom
State/province [16] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Jazz Pharmaceuticals
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol
Statistical analysis plan



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04745026