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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05626959




Registration number
NCT05626959
Ethics application status
Date submitted
15/11/2022
Date registered
25/11/2022
Date last updated
25/11/2022

Titles & IDs
Public title
Evaluating the Efficacy of NTI164 in Young People With Autism Spectrum Disorder
Scientific 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
Secondary ID [1] 0 0
NTIASD2
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autism Spectrum Disorder 0 0
Condition category
Condition code
Mental Health 0 0 0 0
Autistic spectrum disorders
Mental Health 0 0 0 0
Other mental health disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - NTI164

Experimental: NTI164 - 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).

Placebo Comparator: Placebo - 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).


Treatment: Drugs: NTI164
Oil based. Full-spectrum medicinal cannabis plant extract with less than 0.08% THC.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Clinical Global Impression-Severity (CGI-S)
Timepoint [1] 0 0
Baseline, Week 8.
Secondary outcome [1] 0 0
Vineland Adaptive Behaviour Scales, Third Edition
Timepoint [1] 0 0
Baseline, Weeks 16, 28, 40 & 52
Secondary outcome [2] 0 0
Social Responsiveness Scale, 2nd Editions (SRS-2)
Timepoint [2] 0 0
Baseline, Weeks 16, 28, 40 & 52
Secondary outcome [3] 0 0
Clinical Global Impression Scale - Improvement (CGI-I)
Timepoint [3] 0 0
Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Secondary outcome [4] 0 0
Anxiety, Depression and Mood Scale (ADAMS)
Timepoint [4] 0 0
Baseline, Weeks 16, 28, 40 & 52
Secondary outcome [5] 0 0
Sleep Disturbance Scale for Children (SDSC)
Timepoint [5] 0 0
Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Secondary outcome [6] 0 0
Anxiety Scale for Children - Autism Spectrum Disorder
Timepoint [6] 0 0
Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Secondary outcome [7] 0 0
Caregiver Global Impression of Change in Attention (CGI-CA)
Timepoint [7] 0 0
Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52
Secondary outcome [8] 0 0
Caregiver Global Impression of Change (CGI-C) Target Behaviour
Timepoint [8] 0 0
Baseline, Weeks 4, 8, 12, 16, 28, 40 & 52

Eligibility
Key inclusion criteria
INCLUSION CRITERIA:

- 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.

EXCLUSION CRITERIA:

- 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

- 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.
Minimum age
8 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
30/11/2022
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/11/2023
Actual
Sample size
Target
54
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Monash Children's Hospital - Clayton
Recruitment postcode(s) [1] 0 0
3168 - Clayton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Fenix Innovation Group
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Neurotech International
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Monash Health
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05626959
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Michael Fahey, Prof
Address 0 0
Head of Paediatric Neurology
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Kanan Sharma
Address 0 0
Country 0 0
Phone 0 0
+61395946666
Fax 0 0
Email 0 0
MonashChildrensCTC@monashhealth.org
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05626959