NTI164 in Autism Spectrum Disorder

NCT07257939 | Not Yet Recruiting Phase 3

• 2 other identifiers: NTIASD3FENASD3
• Study Type: interventional
• Target: 98
• Locations: 1 country
• Sites: 1

Brief Summary

This is a double-blind, randomised, placebo-controlled study investigating the efficacy of a full-spectrum medicinal cannabis plant extract on core and associated ASD symptoms over placebo. Participants will be randomly allocated to either NTI164 or placebo at a 1:1 ratio and blood samples will be collected and surveys completed at baseline and Week 16. This study will expand efficacy and safety data of NTI164 and provide additional mechanism of action data of NTI164 in this patient cohort.

Conditions

Autism; Autism Spectrum Disorder; Autism Disorder

Keywords

Cannabis; NTI164; Autism spectrum disorder

Outcome Measures

Primary Outcomes (1)

• Social Responsiveness Scale, 2nd Edition (SRS-2)

  Captures overall change in core ASD symptoms across domains including social awareness, social cognition, social communication, social motivation, and restricted and repetitive behaviours. Scores are between 0-195, and higher scores indicate greater impairment.

  Baseline, Week 16

Secondary Outcomes (9)

• Vineland Adaptive Behaviour Scales, 3rd Edition (Vineland-3)

  Baseline, Week 16

• Clinical Global Impression - Improvement (CGI-I)

  Baseline, Week 16

• Clinical Global Impression - Severity (CGI-S)

  Baseline, Week 16

• Anxiety, Depression, and Mood Scale (ADAMS)

  Baseline, Week 16

• Autism Family Experience Questionnaire (AFEQ)

  Baseline, Week 16

Other Outcomes (4)

• Research samples - transcriptomics

  Baseline, Week 16

• Research bloods - proteomics (and phosphoproteomics)

  Baseline, Week 16

• Research bloods - Methylomics

  Baseline, Week 16

Study Arms (2)

Active

EXPERIMENTAL

NTI164

Drug: NTI164

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

NTI164 DRUG

A CBDA-dominant full-spectrum medicinal cannabis plant extract with extremely low THC.

Also known as: FEN164

Active

Placebo DRUG

Placebo oil suspension

Placebo

Eligibility Criteria

• Age: 6 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participant is aged 6 years to 25 years (inclusive).
• Participant is at a healthy weight at the discretion of the Principal Investigator.
• Written informed consent from parent or legal guardian according to the local law.
• 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 II or III ASD confirmed by a validated assessment tool.
• All treatments including medications and therapies for ASD related symptoms must have been stable for 12 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.

You may not qualify if:

• 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., 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, vasectomised 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 jeopardise 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 12 weeks or at screening or randomisation.

Sponsors & Collaborators

• Fenix Innovation Group: lead
• Neurotech International Limited: collaborator

Study Sites (1)

Monash Health

Clayton, Victoria, 3168, Australia

Location

MeSH Terms

Conditions

Autistic Disorder; Autism Spectrum Disorder; Marijuana Abuse

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders; Substance-Related Disorders; Chemically-Induced Disorders

Central Study Contacts

Kanan Sharma

CONTACT

+61395946666; monashchildrensctc@monashhealth.org

Michael C Fahey

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: All study team members will be blinded during the main treatment phase of this study (i.e up to Week 16). Only Clinical Trial Pharmacy staff will be unblinded.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double-blind, randomised, placebo-controlled, parallel

Study Record Dates

• First Submitted: November 17, 2025
• First Posted: December 2, 2025
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2030
• Last Updated: December 2, 2025

Record last verified: 2025-11

Locations

AU (1)