Investigating the effects of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on paediatric acute-onset neuropsychiatric syndrome (PANS): An open-label study.
Investigating the effects of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on gene regulation and immune dysfunction in paediatric acute-onset neuropsychiatric syndrome: An open-label study.
Fenix Innovation Group
15 participants
May 10, 2023
Interventional
Conditions
Summary
This is an 18 to 54-week open-label study to evaluate the efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the severity of paediatric acute-onset neuropsychiatric syndrome (PANS) in children. The purpose of this study is to determine how effective NTI164 is in patients with PANS when treated for 18 to 54 weeks. Participants will commence treatment with a daily dose of 5mg/kg of NTI164. 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). Participants will continue to receive their respective maximum dose for eight (8) weeks (Treatment phase). Participants who wish to continue receiving their maximum tolerated dose beyond the 8-week Treatment phase may do so for up to fifty-four (54) weeks (Extension phase). At the end of the Treatment or Extension phase, participants will be gradually decreased by 5 mg/kg for a period of 4 weeks until the end of their participation (Down-titration phase). Efficacy will be measured and monitored by performing participant- and psychologist- led questionnaires specific to measuring changes in the emotions and behaviour of patients with PANS. Whole blood RNA sequencing will validate the immune dysfunction signature.
Eligibility
Inclusion Criteria7
- - 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
Exclusion Criteria8
- 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)
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Interventions
Full-spectrum medicinal cannabis plant extract with 0.08% THC (NTI164). NTI164 is an oil that will be administered orally over the course of the study. The study involves the following phases: • Baseline/Up-titration phase: Children will receive a baseline dose of 5mg/kg/day of NTI164 that will be increased weekly by 5mg/kg for a period of 4 weeks until the maximum tolerated dose or 20mg/kg is achieved. • Treatment phase: Children will receive the maximum tolerated dose daily or 20mg/kg/day for either an 8-week period or they may choose to extend this up to 54 weeks (Extension phase). • Down-titration phase: At the end of the Treatment Phase, children will receive a dosage that will be gradually decreased by 5mg/kg/week for a period of 4 weeks until the end of the study. • Extension phase: Participants who choose to continue receiving the maximum tolerated dose beyond the set 8 weeks may do so for up to 54 weeks. They will undergo the Down-titration phase at the end of their Extension phase. The minimum participation duration time is 18 weeks and the maximum duration time is 54 weeks inclusive of the 8 week down titration phase. Adherence to intervention will be monitored by drug product return accountability, completion of online drug administration forms and study-specific questionnaires.
Locations(2)
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ACTRN12622001419752