Evaluating the Efficacy of NTI164 in Young People With Autism Spectrum Disorder

NCT05626959 | Unknown Phase 2

• 1 other identifier: NTIASD2
• Study Type: interventional
• Target: 54
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline, Week 8.

Secondary Outcomes (8)

• Vineland Adaptive Behaviour Scales, Third Edition

  Baseline, Weeks 16, 28, 40 & 52

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

  Baseline, Weeks 16, 28, 40 & 52

• Clinical Global Impression Scale - Improvement (CGI-I)

  Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52

• Anxiety, Depression and Mood Scale (ADAMS)

  Baseline, Weeks 16, 28, 40 & 52

• Sleep Disturbance Scale for Children (SDSC)

  Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52

Study Arms (2)

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).

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).

Drug: NTI164

Interventions

NTI164 DRUG

Oil based. Full-spectrum medicinal cannabis plant extract with less than 0.08% THC.

Also known as: Full-spectrum medicinal cannabis plant extract 0.08% THC

NTI164; Placebo

Eligibility Criteria

• Age: 8 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

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., 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
• 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

Sponsors & Collaborators

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

Study Sites (1)

Monash Children's Hospital

Clayton, Victoria, 3168, Australia

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Dronabinol

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Michael Fahey, Prof

  Head of Paediatric Neurology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kanan Sharma

CONTACT

+61395946666; MonashChildrensCTC@monashhealth.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Allocation is concealed utilising central randomisation by computer.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: November 15, 2022
• First Posted: November 25, 2022
• Study Start: November 30, 2022
• Primary Completion: March 1, 2023
• Study Completion: November 30, 2023
• Last Updated: November 25, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)