Safety and Efficacy of Oral Full-Spectrum Medicinal Cannabis Plant Extract in Children With Autism Spectrum Disorder.

Fenix Innovation Group

Status and phase: Active, not recruiting; Phase 2; Phase 1

Conditions: Autism Spectrum Disorder

Treatments: Drug: FEN164

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT05516407

FNX001-21

Details and patient eligibility

About

This is a 20-week open-label study to evaluate the safety and efficacy of full-spectrum medicinal cannabis plant extract < 0.08% THC (FEN164) in children with Autism Spectrum Disorder.

Full description

The study population will include twenty (20) boys and girls aged eight (8) years through to seventeen (17) years that have a medical diagnosis of Level 2 and 3 Autism Spectrum Disorder (ASD) as confirmed by the Autism Diagnostic Observational Schedule (ADOS-2) criteria.

Participants will commence treatment with a daily dose of 5mg/kg of FEN164. This will gradually increase over a four-week period until the maximum tolerated daily dose or 20mg/kg per day is achieved (Up-titration phase/Stage 1). Participants will continue to receive their respective maximum dose for eight (8) weeks (Treatment phase/Stage 2) which will be gradually decreased by 5 mg/kg for a period of 4 weeks until the end of the study (Down-titration phase).

Enrollment: 18 patients

Sex: All

Ages: 8 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participant is aged 8 years to 17 years (inclusive)
• Participant is at a healthy weight at the discretion of the Principal Investigator.
• Parents or caregivers can give informed consent for participation in the trial with assent from individuals with autism.
• Participants can comply with trial requirements.
• According the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) criteria the participant has a diagnosis of Level 2 or 3 Autism Spectrum Disorder (ASD) confirmed by Autism Diagnostic Observational Schedule (ADOS-2) criteria
• All treatments including medications and therapies for ASD related symptoms must have been stable for 4 weeks before enrolment and for the duration of the trial wherever possible.
• Participants must be able to swallow liquid.
• Consent giver must be able to understand the requirements of the study.

Exclusion criteria

• Current diagnosis of bipolar disorder, psychosis, schizophrenia, schizoaffective disorder, or active major depression
• Has a diagnosis other than ASD that dominates the clinical presentation (e.g., Attention Deficit Hyperactivity Disorder [ADHD])
• Has a degenerative condition
• Changes in anticonvulsive therapy within the last 12 weeks
• Taking omeprazole, lansoprazole, tolbutamide, warfarin, sirolimus, everolimus, temsirolimus, tacrolimus, clobazam, 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
• 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 with 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 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 behaviour (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 enrolled into this trial.
• Participant has plans to travel outside their country of residence during the trial, unless the participant has confirmation that the product is permitted in the destination country/state

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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18 participants in 1 patient group

FEN164

Experimental group

Description:

Full-Spectrum Medicinal Cannabis Plant Extract with less than 0.08% THC (FEN164) Stage 1: 5mg/kg, 10mg/kg, 15mg/kg, 20mg/kg (1 week each) Stage 2: 20mg/kg (8 weeks), 15mg/kg, 10mg/kg, 5mg/kg (1 week each)

Treatment:

Drug: FEN164