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PANS Pediatric Acute-Onset Neuropsychiatric SyndromePhase 1/2CannaPANSNCT06621888

Investigating The Effects Of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) On Paediatric Acute-onset Neuropsychiatric Syndrome (PANS)

A Phase 1/2 study testing NTI164 in people with PANS Pediatric Acute-Onset Neuropsychiatric Syndrome, run by Fenix Innovation Group at 2 sites in Australia.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study investigates the effectiveness of a medicinal cannabis extract (NTI164) with 0.08% THC in treating children with pediatric acute-onset neuropsychiatric syndrome (PANS) over a period of 18 to 54 weeks. Read more about this study

Who is it for?
Ages 1 year to 17 years
What is being tested?
NTI164
Could I receive a placebo?
No, everyone receives an active treatment
How many people
18 participants
Where
2 sitesAustralia
Study dates
2 Jun 2023to 1 Dec 2025
Phase
Phase 1/2
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 1 year to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 1 - 17 years of age
  • Patients who fulfil PANS criteria
  • Acute onset of OCD or severely restricted food intake
  • Concurrent presentation of additional neuropsychiatric symptoms from at least 2 of the following 7 categories: anxiety, emotional lability/depression, irritability, aggression or severely oppositional behaviours, behavioural regression, deterioration in school performance, sensory or motor abnormalities (e.g. tics), somatic symptoms (e.g. sleep disturbances, enuresis or increase in urinary frequency)
  • Symptoms not better explained by a known neurologic or medical disorder (e.g. Sydenham's chorea)
  • RCADS-P scores of \>65 (a scale of anxiety, social phobia, panic disorder, OCD, and low mood, a score of \>65 infers moderate-significant impairment)
  • Other patient medications (e.g. anti-psychotics) must be stable for at least 12 weeks prior to trial participation

Who cannot join

  • Infection and/or antibiotic use in the 2 weeks prior to trial participation (i.e. baseline blood tests and commencement of NTI164)
  • Recent changes to other patient medication (e.g. addition or escalation of anxiolytics, anti-depressants etc; medication dosage must be stable for at least 12 weeks prior to trial participation)
  • Intellectual disability preventing adequate assent from patient, or that would affect reporting throughout trial; patients with intellectual disability must still have the capacity to verbalise their symptoms/experiences
  • Ongoing immunomodulating or immunosuppressive treatment use in the previous 12 weeks, including steroids, IVIG, antibiotics, low-dose naltrexone, mycophenolate, Rituximab etc.
  • Currently using or has used recreational or medicinal cannabis or cannabinoid-based medications (e.g. Sativex ®, Epidiolex ®) in the previous 12 weeks and/or is unwilling or unable to abstain for the duration of the trial
  • Underlying renal impairment, cardiovascular issues (e.g. arrhythmia), current or previous thrombosis
  • Impaired hepatic function, defined as serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 2 x upper limit of normal (ULN) or total bilirubin (TBL) \> 2 x ULN; this criterion can only be confirmed once baseline laboratory results are available and participants who fail this criterion will not proceed in this study
  • Other diagnosed neurological condition likely to be contributing to OCD/neuropsychiatric symptoms (e.g. Huntington's disease)

Trial locations and contacts

2 sites in 1 country.

Australia2 sites
  • Monash Children's Hospital

    Melbourne, Victoria

  • The Childrens Hospital at Westmead

    Sydney, New South Wales, 2145

Other trials in Australia

About this study

This study investigates the effectiveness of a medicinal cannabis extract (NTI164) with 0.08% THC in treating children with pediatric acute-onset neuropsychiatric syndrome (PANS) over a period of 18 to 54 weeks. Participants, aged 18 to 54, will start with a daily dose of 5mg/kg, gradually increasing to a maximum of 20mg/kg over four weeks. After reaching their maximum tolerated dose, they will maintain this dose for eight weeks, with an option to extend up to 54 weeks. The study will measure the treatment\'s efficacy using questionnaires on emotional and behavioral changes, and verify the results with whole blood RNA sequencing to assess immune dysfunction.

Read the detailed description

This open-label study spans 18 to 54 weeks and aims to assess the efficacy of Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164) for treating pediatric acute-onset neuropsychiatric syndrome (PANS) in children. The main objective is to evaluate how effective the NTI164 treatment is over the specified duration.

The study protocol involves several phases:

  1. Up-titration phase: Participants will start with a daily dosage of 5 mg/kg of NTI164, incrementally increasing this dose over a four-week period until reaching either the maximum tolerated dose or 20 mg/kg per day.
  2. Treatment phase: Following the up-titration, participants will continue at their maximum tolerated dose for eight weeks.
  3. Extension phase: After the initial eight-week treatment phase, participants have the option to continue at their maximum dose for up to an additional 46 weeks, totaling a possible 54 weeks of treatment.
  4. Down-titration phase: At the conclusion of the treatment or extension phases, the dosage will be gradually reduced by 5 mg/kg over four weeks until the participant\'s involvement in the study ends.

The effectiveness of NTI164 will be monitored through both participant- and psychologist-led questionnaires, which are designed to track changes in the emotions and behavior of the patients with PANS. Additionally, the study will employ whole blood RNA sequencing as a method to validate the presence of an immune dysfunction signature, aiming to provide a biomarker for response to treatment. This comprehensive approach seeks to ensure a thorough evaluation of NTI164's potential benefits in alleviating the symptoms of PANS.

Being tested
NTI164
Medical subject headings
Pediatric acute-onset neuropsychiatric syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • NTI164Drug

    This intervention uses Full-Spectrum Medicinal Cannabis Plant Extract with a low THC concentration of 0.08% (NTI164), specifically formulated to treat pediatric acute-onset neuropsychiatric syndrome (PANS). The dosing regimen is carefully structured to increase from an initial 5 mg/kg per day up to a maximum of 20 mg/kg, tailored to individual tolerance levels. This gradual titration and the option to extend treatment up to 54 weeks distinguishes it from other interventions that may use different concentrations of THC or shorter treatment durations. The efficacy of NTI164 is rigorously assessed through psychological evaluations and biomarker analyses.

Study groups in NCT06621888
Study groupRoleWhat they receive
Active NTI164 GroupExperimentalParticipants in this group receive Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164) to treat pediatric acute-onset neuropsychiatric syndrome (PANS). The treatment begins with an up-titration phase where doses start at 5 mg/kg daily and increase to a maximum of 20 mg/kg. This is followed by an 8-week treatment phase at the maximum tolerated dose. Participants have the option to extend this phase up to 54 weeks. The study concludes with a down-titration phase, gradually reducing the dose over 4 weeks. Efficacy is assessed through psychological questionnaires and immune function tests.
Receives: Drug: NTI164

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Clinical Global Impression Scale-Improvement

    The CGI-I (Clinical Global Impressions - Improvement) scale rates patient improvement on a scale from 1 to 7. Lower scores indicate better improvement.

    Measured: Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment. Additional timepoints for Extension phase: Weeks 28, 40, 52 post-commencement of treatment.

  2. Revised Childrens Anxiety and Depression Scale-Parent Version

    The RCADS-P (Revised Child Anxiety and Depression Scale - Parent Version) assesses anxiety and depression in children, with individual item scores ranging from 0 to 3. Higher scores indicate more severe symptoms, meaning lower scores suggest better emotional well-being.

    Measured: Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment. Additional timepoints for Extension phase: Weeks 28, 40, 52 post-commencement of treatment.

Secondary outcomes (5)
  1. Yale Global Tic Severity Scale

    The Yale Global Tic Severity Scale (YGTSS) measures the severity of tics in individuals, with individual item scores ranging from 0 to 5 for both motor and vocal tics. Higher scores indicate more severe tics and lower scores reflect better tic control.

    Measured: Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment. Additional timepoints for Extension phase: Weeks 28, 40, 52 post-commencement of treatment.

  2. Children's Yale-Brown Obsessive-Compulsive Scale

    The Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) assesses the severity of obsessive-compulsive symptoms in children, with individual item scores ranging from 0 to 4. Higher scores indicate more severe symptoms and lower scores suggest better symptom control.

    Measured: Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment. Additional timepoints for Extension phase: Weeks 28, 40, 52 post-commencement of treatment.

  3. Conners Scale

    The Conners\' Scale measures behavioural problems, with individual item scores ranging from 0 to 3. Higher scores indicate more severe behavioural issues and lower scores reflect better behavioural functioning.

    Measured: Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment. Additional timepoints for Extension phase: Weeks 28, 40, 52 post-commencement of treatment.

  4. EQ-5D-Y

    The EQ-5D-Y (EuroQol Five-Dimension Youth) is a measure of health-related quality of life in children and adolescents. It assesses five dimensions, with individual item scores ranging from 1 to 3. A higher score indicates more health-related difficulties, meaning lower scores reflect better quality of life.

    Measured: Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment. Additional timepoints for Extension phase: Weeks 28, 40, 52 post-commencement of treatment.

  5. Blood Transcriptomic Signature

    The Blood Transcriptomic Signature outcome assesses changes in gene expression in PANS children post-NTI164 treatment, compared to baseline and controls. RNA sequencing of blood samples will identify modifications in the transcriptomic profile, providing insights into the treatment's impact on molecular processes.

    Measured: Baseline (pre-dose) and 16 weeks post-commencement of treatment.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Oct 2024
  2. Study start2 Jun 2023
  3. Primary completion1 Dec 2025
  4. Study completion1 Dec 2025

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
18 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06621888
Sponsor's protocol ID
NTIPANS1
Other IDs
ACTRN12622001419752
Acronym
CannaPANS
Status last verified
Sep 2026
First submitted
11 Sep 2024
Last updated
9 Sep 2026
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No: IPD will not be shared as per commercial in confidence restrictions.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing NTI164 in people with PANS Pediatric Acute-Onset Neuropsychiatric Syndrome, run by Fenix Innovation Group at 2 sites 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 7 inclusion and 8 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 9 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 2 sites 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Clinical Global Impression Scale-Improvement (time frame: Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment. Additional timepoints for Extension phase: Weeks 28, 40, 52 post-commencement of treatment.). The study lists 2 primary outcomes in all. 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 9 Sep 2026.

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