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

NCT06621888 | Active Not Recruiting Phase 1

• 2 other identifiers: NTIPANS1ACTRN12622001419752
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 2

Brief 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. 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.

Conditions

PANS Pediatric Acute-Onset Neuropsychiatric Syndrome

Outcome Measures

Primary Outcomes (2)

• 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.

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

• 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.

  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)

• Yale Global Tic Severity Scale

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

• Children's Yale-Brown Obsessive-Compulsive Scale

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

• Conners Scale

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

• EQ-5D-Y

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

• Blood Transcriptomic Signature

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

Study Arms (1)

Active NTI164 Group

EXPERIMENTAL

Participants 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.

Drug: NTI164

Interventions

NTI164 DRUG

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.

Active NTI164 Group

Eligibility Criteria

• Age: 1 Year - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

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

Study Sites (2)

The Childrens Hospital at Westmead

Sydney, New South Wales, 2145, Australia

Location

Monash Children's Hospital

Melbourne, Victoria, Australia

Location

MeSH Terms

Conditions

Pediatric acute-onset neuropsychiatric syndrome

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 11, 2024
• First Posted: October 1, 2024
• Study Start: June 2, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: October 1, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2)