A Safety and Efficacy Study of GWP42003-P (Cannabidiol; CBD) in Children and Young Adults with Autism Spectrum Disorder.

Phase 1

Conditions: Autism Spectrum Disorder (ASD)
MedDRA version: 21.1Level: PTClassification code 10063844Term: Autism spectrum disorderSystem Organ Class: 10037175 - Psychiatric disorders
Therapeutic area: Psychiatry and Psychology [F] - Mental Disorders [F03]

Registration Number: EUCTR2020-002819-21-ES

Lead Sponsor: GW Research Ltd

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 160

Inclusion Criteria

For inclusion in the trial, patients must fulfill ALL of the following criteria:
• Male or female aged 6 to 17 years (inclusive).
• Patient weight is at least 12 kg.
• Patients (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.
• Patient and their caregiver are willing and able (in the investigator’s opinion) to comply with all trial requirements.
• Patient has a diagnosis of ASD as per DSM-5 criteria for ASD, confirmed by 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 ADI-R at screening.
• CGI-S > or = 4 (moderately ill) at screening and randomization.
• ABC-I subscale score = 15 at screening.
• IQ > or = 70 at screening, or measured within 1 year of screening, using 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.
• Patients must have the ability to swallow the IMP, provided as a liquid solution.
• Patient 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.
• Patient and/or parent(s)/legal representative is/are willing to allow the patient’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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 160
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The patient may not enter the trial if ANY of the following apply:
• Current diagnosis of bipolar disorder, psychosis, schizophrenia, schizoaffective disorder, or major depression (patients with depression in remission may be included).
• Has a diagnosis other than ASD that dominates the clinical presentation (e.g., 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 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®/Epidyolex®) within the 12 weeks prior to screening and is unwilling to abstain for the duration of the trial.
Patient has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the IMP, such as sesame oil.
• Patient has moderately impaired hepatic function at screening, defined as serum ALT or AST > 2 × ULN or TBL > 2 × ULN’
This criterion can only be confirmed once the laboratory results are available; patients enrolled into the trial who are later found to meet this criterion must be screen-failed.
• Patient 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.
• Patient is female and of childbearing potential (i.e., following menarche and until becoming postmenopausal for > o = 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 patient who is pregnant (positive pregnancy test), lactating or planning pregnancy during the course of the trial or within 12 weeks thereafter.
• Patient has received an IMP within the 12 weeks prior to the screening visit.
• Patient had brain surgery or traumatic brain injury within 1 year of screening.
• Patient has any other significant disease or disorder which, in the opinion of the investigator, may either put the patient, other participants, or site staff at risk because of participation in the trial, may influence the result of the trial, or may affect the patient’s ability to take part in the trial.
• Any abnormalities identified following a physical examination of the patient that, in the opinion of the investigator, would jeopardize the safety of the patient if they took part in the trial.
• Any history of suicidal behavior (lifelong) or any suicidal ideation of type 4 or 5 on the C-SSRS in the last 4 weeks or at screening or randomization.
Patient has donated blood during the past 12 weeks and is unwilling to abstain from donation of blood during the trial.
• Patient 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).
Patient has previously been randomized into this trial.
• Patient has plans to

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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