Autism Spectrum DisorderPhase 2/3NCT05626959
Evaluating the Efficacy of NTI164 in Young People With Autism Spectrum Disorder
A Phase 2/3 study testing NTI164 in people with Autism Spectrum Disorder, run by Fenix Innovation Group at 1 site in Australia.
Trial summary
This is an 18 to 54 week study assessing the efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the severity of autism spectrum disorder in young people. Read more about this study
- Who is it for?
- Ages 8 to 17 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- NTI164
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 54 participants
- Where
- 1 siteAustralia
- Study dates
- 30 Nov 2022to 30 May 2026
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Show all 19Show fewer
- 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 greater than or equal to 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 locations and contacts
1 site in 1 country.
About this study
Official title: A Phase II/III Double-Blind, Randomised and Controlled-to-Open-Label Study Assessing the Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the Severity of Autism Spectrum Disorder in Young People
This is an 18 to 54 week study assessing the efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the severity of autism spectrum disorder in young people.
Read the detailed description
The purpose of this study is to determine the effectiveness of NTI164 in patients with ASD when treated with 20mg/kg/day for 8 - 54 weeks.
The study comprises of an 8-week double-blinded randomised controlled treatment period followed by an 8-week open-label maintenance period followed by a 2-week wash-out period. Participants who wish to continue receiving the study treatment beyond the 16 week period may do so for up to fifty-two weeks (Extension phase).
Efficacy will be measured and monitored by performing participant- and psychologist- led questionnaires specific to measuring changes in the behaviour of patients with ASD.
Safety will be measured and monitored by performing full blood examinations and liver and renal function tests throughout the study.
Additional assessments include microbiome and inflammatory marker assessments.
- Conditions
- Autism Spectrum Disorder
- Being tested
- NTI164
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NTI164Drug
Also called Full-spectrum medicinal cannabis plant extract 0.08% THC
Oil based. Full-spectrum medicinal cannabis plant extract with less than 0.08% THC.
| Study group | Role | What they receive |
|---|---|---|
| NTI164 | Experimental | Full-Spectrum Medicinal Cannabis Plant Extract with less than 0.08% THC (NTI164)
Randomised Controlled Phase:
Part A: 5mg/kg, 10mg/kg, 15mg/kg, 20mg/kg (1 week each, total duration = 4 weeks) Part B: 20mg/kg or maximum tolerated dose (total duration = 4 weeks).
Open-Label Phase 20mg/kg or maximum tolerated dose (total duration = 8 weeks).
Extension Phase 20mg/kg or maximum tolerated dose (total duration = 36 weeks). Receives: Drug: NTI164 |
| Placebo | Placebo comparator | Randomised Controlled Phase:
Part A: 5mg/kg, 10mg/kg, 15mg/kg, 20mg/kg (1 week each, total duration = 4 weeks) Part B: 20mg/kg or maximum tolerated dose (total duration = 4 weeks). Receives: Drug: NTI164 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Clinical Global Impression-Severity (CGI-S)
Reflects clinician's impression of severity of illness on a 7-point scale ranging from 1=not at all to 7=among the most extremely ill.
Measured: Baseline, Week 8.
Secondary outcomes (8)
Vineland Adaptive Behaviour Scales, Third Edition
Used to measure adaptive functioning across three core domains (Communication, Daily Living Skills, and Socialization), and two optional domains (Motor Skills and Maladaptive Behaviour); items are rated on a 3-point scale (0=never; 1=sometimes; 2=usually or often). The core domains sum to a total Adaptive Behaviour Composite.
Measured: Baseline, Weeks 16, 28, 40 & 52
Social Responsiveness Scale, 2nd Editions (SRS-2)
Five domains are assessed including: Social Awareness, Social Cognition, Social Communication, Social Motivation, and Restricted Interests and Repetitive Behaviour. Items are scored on a 4-point scale (ranging from 1=not true to 4=almost always true).
Measured: Baseline, Weeks 16, 28, 40 & 52
Clinical Global Impression Scale - Improvement (CGI-I)
This is a 7-point scale measuring symptom change from baseline.
Measured: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Anxiety, Depression and Mood Scale (ADAMS)
28 symptom items that resolve into five subscales labelled: Manic/Hyperactive Behaviour, Depressed Mood, Social Avoidance, General Anxiety, and Compulsive Behaviour. Items are rated on 4-point scale ranging from 0=not a problem to 3=severe problem.
Measured: Baseline, Weeks 16, 28, 40 & 52
Sleep Disturbance Scale for Children (SDSC)
Six subscales including Disorders of Initiating and Maintaining Sleep, Sleep Breathing Disorders, Disorders of Arousal, Sleep Wake Transition Disorders, Disorders of Excessive Somnolence, and Sleep Hyperhydrosis. Items are rated on 5-point scale where 1=never and 5=always (daily). Subscale scores sum to equal a total score
Measured: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Anxiety Scale for Children - Autism Spectrum Disorder
A form developed to detect symptoms of anxiety in youth with ASD. Composed of four subscales (Performance Anxiety, Uncertainty, Anxious Arousal, and Separation Anxiety), items are rated on a 4-point scale (0=never and 3=always). Subscales sum to equal a total score.
Measured: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Caregiver Global Impression of Change in Attention (CGI-CA)
Reflects clinician's impression of change in attention on a 7-point scale ranging from 1=not at all to 7=very severe problem. Provided as Baseline and Post-Baseline questionnaires.
Measured: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Caregiver Global Impression of Change (CGI-C) Target Behaviour
Reflects clinician's impression of change of behaviour on a 7-point scale ranging from 1=not at all to 7=very severe problem. Provided as Baseline and Post-Baseline questionnaires.
Measured: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Nov 2022
- Study start30 Nov 2022
- Primary completion1 Mar 2024
- Study completion30 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- At the beginning of the study, participants will be randomised into either the active group or placebo group. Both groups will commence a double-blinded baseline dose of either 5mg/kg/day of NTI164 or Placebo that will be increased weekly by 5mg/kg for a period of 4 weeks until the maximum tolerated dose or 20/mg/kg/day is achieved. At the end of the 8-week period, both study groups will be unblinded and all participants will begin or continue NTI164 for 8 weeks.
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 54 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fenix Innovation Group
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Neurotech International Limited, Monash Health
- Responsible party
- Sponsor
- Investigators
- Michael Fahey, Prof, Principal investigator, Head of Paediatric Neurology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05626959
- Sponsor's protocol ID
- NTIASD2
- Status last verified
- Sep 2026
- First submitted
- 15 Nov 2022
- Last updated
- 10 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II/III Double-Blind, Randomised and Controlled-to-Open-Label Study Assessing the Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the Severity of Autism Spectrum Disorder in Young People
- ClinicalTrials.gov ID
- NCT05626959
- Acronym / sponsor ID
- NTIASD2
- Conditions
- Autism Spectrum Disorder
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 54 participants (actual)
- Lead sponsor
- Fenix Innovation GroupIndustry (pharma, biotech, devices)
- Collaborators
- Neurotech International Limited, Monash Health
- Study start
- 30 Nov 2022 (actual)
- Primary completion
- 1 Mar 2024 (actual)
- Study completion
- 30 May 2026 (actual)
- First posted
- 25 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an 18 to 54 week study assessing the efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the severity of autism spectrum disorder in young people.
Detailed description
The purpose of this study is to determine the effectiveness of NTI164 in patients with ASD when treated with 20mg/kg/day for 8 - 54 weeks.
The study comprises of an 8-week double-blinded randomised controlled treatment period followed by an 8-week open-label maintenance period followed by a 2-week wash-out period. Participants who wish to continue receiving the study treatment beyond the 16 week period may do so for up to fifty-two weeks (Extension phase).
Efficacy will be measured and monitored by performing participant- and psychologist- led questionnaires specific to measuring changes in the behaviour of patients with ASD.
Safety will be measured and monitored by performing full blood examinations and liver and renal function tests throughout the study.
Additional assessments include microbiome and inflammatory marker assessments.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- At the beginning of the study, participants will be randomised into either the active group or placebo group. Both groups will commence a double-blinded baseline dose of either 5mg/kg/day of NTI164 or Placebo that will be increased weekly by 5mg/kg for a period of 4 weeks until the maximum tolerated dose or 20/mg/kg/day is achieved. At the end of the 8-week period, both study groups will be unblinded and all participants will begin or continue NTI164 for 8 weeks.
| Arm | Type | Receives |
|---|---|---|
| NTI164 | Experimental | Drug: NTI164Full-Spectrum Medicinal Cannabis Plant Extract with less than 0.08% THC (NTI164) Randomised Controlled Phase: Part A: 5mg/kg, 10mg/kg, 15mg/kg, 20mg/kg (1 week each, total duration = 4 weeks) Part B: 20mg/kg or maximum tolerated dose (total duration = 4 weeks). Open-Label Phase 20mg/kg or maximum tolerated dose (total duration = 8 weeks). Extension Phase 20mg/kg or maximum tolerated dose (total duration = 36 weeks). |
| Placebo | Placebo comparator | Drug: NTI164Randomised Controlled Phase: Part A: 5mg/kg, 10mg/kg, 15mg/kg, 20mg/kg (1 week each, total duration = 4 weeks) Part B: 20mg/kg or maximum tolerated dose (total duration = 4 weeks). |
Who may qualify
- Ages
- 8 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (19 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NTI164Also: Full-spectrum medicinal cannabis plant extract 0.08% THCOil based. Full-spectrum medicinal cannabis plant extract with less than 0.08% THC. | Drug | NTI164, Placebo |
Primary outcome
Change in Clinical Global Impression-Severity (CGI-S)
Time frame: Baseline, Week 8.
Reflects clinician's impression of severity of illness on a 7-point scale ranging from 1=not at all to 7=among the most extremely ill.
8 secondary outcomes
Vineland Adaptive Behaviour Scales, Third Edition
Time frame: Baseline, Weeks 16, 28, 40 & 52
Social Responsiveness Scale, 2nd Editions (SRS-2)
Time frame: Baseline, Weeks 16, 28, 40 & 52
Clinical Global Impression Scale - Improvement (CGI-I)
Time frame: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Anxiety, Depression and Mood Scale (ADAMS)
Time frame: Baseline, Weeks 16, 28, 40 & 52
Sleep Disturbance Scale for Children (SDSC)
Time frame: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Anxiety Scale for Children - Autism Spectrum Disorder
Time frame: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Caregiver Global Impression of Change in Attention (CGI-CA)
Time frame: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Caregiver Global Impression of Change (CGI-C) Target Behaviour
Time frame: Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michael Fahey, Prof principal investigator
Head of Paediatric Neurology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing NTI164 in people with Autism Spectrum Disorder, run by Fenix Innovation Group at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Clinical Global Impression-Severity (CGI-S) (time frame: Baseline, Week 8.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.