Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12621000370808
Ethics application status
Approved
Date submitted
25/01/2021
Date registered
31/03/2021
Date last updated
31/03/2021
Date data sharing statement initially provided
31/03/2021
Type of registration
Prospectively registered

Titles & IDs
Public title
Observational Study of two low dose medicinal cannabis oral oils to treat Autism associated behaviours.
Scientific title
An open label, observational study to investigate the tolerance and response to two low dose medicinal cannabis products with different THC:CBD ratios in Autism Spectrum Disorder (ASD) patients
Secondary ID [1] 303260 0
ZTL-105
Universal Trial Number (UTN)
Trial acronym
Hope ASD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autism Spectrum Disorder (ASD) 320439 0
Condition category
Condition code
Mental Health 318335 318335 0 0
Autistic spectrum disorders

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
This is an open label study enrolling people (including children and adolescents (from 6 to 18 years) and adults) with Autism Spectrum Disorder (ASD) presenting with behavioural symptoms that receive medicinal cannabis as prescribed per standard practice. Patients who are receiving HOPE 1 and/or HOPE 2 products as part of their standard of care treatment (through the Special Access or Authorised Prescriber schemes) will be offered the option to consent to the observational study. This study provides the sponsor with patient data assessed for their disease and safety according to standard practice. HOPE 1 and HOPE 2 are oral oil formulations, administered twice daily at a dosage recommended by the study doctor as per their standard of care practice. Patient's may receive HOPE 1 and/or HOPE 2. It is recommended that starting doses should be low, and increased over time in 0.25ml increments or as tolerated. Each patient will be observed for a period of six months from their first visit. Visits will be completed by their treating doctor (General Practitioner), and patients will be monitored for adverse events and adherence to dosing. Each visit shall take approximately 30 minutes. Patients and/or carer as applicable will complete the following ASD related standard of care questionnaires approximately monthly or as directed by their doctor: (Aberrant Behaviour Checklist (ABC), Clinical Global Impression (CGI), Caregiver Global Impression of Change (CaGI), Sleep Disturbance Scale (SDSC), Autism Parenting Stress Index (APSI) and Insomnia Severity Index (ISI - adults only). The following assessments will be collected approximately monthly or as directed by their treating doctor: collection of vital signs and review of adverse effects. Standard of care blood tests (if required) may be collected approximately 3 monthly, or as directed by the treating doctor. Data collected from these visits will form the basis of the data analysis for the study.
Intervention code [1] 319560 0
Not applicable
Comparator / control treatment
N/A observational study. No control group.
Control group
Uncontrolled

Outcomes
Primary outcome [1] 326303 0
The primary objective of this study is to investigate the effect of prescribed medicinal cannabis products HOPE 1 and HOPE 2 on a composite of behavioural symptoms of ASD in people based on standard of care assessments

This is assessed by completion of the following ASD related standard of care assessments: Approximately monthly collection of vital signs and review of adverse effects. Standard of care blood tests (if required) may be collected approximately 3 monthly, or as directed by the treating doctor. The following questionnaires will be completed approximately monthly or as directed by their doctor: (Aberrant Behaviour Checklist (ABC), Clinical Global Impression (CGI), Caregiver Global Impression of Change (CaGI), Sleep Disturbance Scale (SDSC), Autism Parenting Stress Index (APSI) and Insomnia Severity Index (ISI - adults only).
Timepoint [1] 326303 0
Patient data is collected monthly for 6 months post starting the trial. Last data collection expected Jan 2022
Secondary outcome [1] 390988 0
To investigate and compare the effect of HOPET 1 and HOPE 2 on Parental Stress as per questionnaire Autism Parenting Stress Index
Timepoint [1] 390988 0
Patient data is collected for 6 months post starting the trial. Last data collection expected Jan 2022
Secondary outcome [2] 390989 0
To investigate and compare the effect of HOPE 1 and HOPE 2 on sleep in people with ASD; as per Sleep Disturbance Scale Questionnaire and Insomnia Severity Index
Timepoint [2] 390989 0
Patient data is collected monthly for 6 months post starting the trial. Last data collection expected Jan 2022
Secondary outcome [3] 390990 0
To investigate the susceptibility to adverse effects in people with ASD prescribed medicinal cannabis products HOPE 1 and HOPE 2 as per standard of care review by treating doctor.
Timepoint [3] 390990 0
Patient data is collected monthly for 6 months post starting the trial. Last data collection expected Jan 2022

Eligibility
Key inclusion criteria
a. Signed consent (patients and/or carer)
b. Have a documented history of Autism Spectrum Disorder (including diagnosis and severity of ASD)
c. Patient and caregiver must be able to return to clinic on days specified in the protocol
d. Patient and caregiver must be able to adhere to all assessments as described
e. Have a valid assessment of severity using the Connors Parent and School assessment or similar tool within the previous 12 months
f. Have exhausted conventional therapies listed on the ARTG for ASD
g. For paediatric patients only: Have trialled or are being considered for treatment with antipsychotic medications
h. For paediatric patients only: Have a letter of support or referral from their treating paediatrician to trial cannabinoid therapies or be managed by Emerald Clinics.
i. Planned to be administered either HOPETM 1 and/or HOPETM 2 medicinal cannabis
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
j. Patient is currently using recreational or medicinal cannabis, or synthetic cannabinoid-based medications within one month prior to study entry and is unwilling to abstain for the duration of the trial.

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Defined population
Timing
Prospective
Statistical methods / analysis
There is no statistical sample size determination for this early phase study. The sample size is based on medical and practical grounds rather than statistical grounds. Descriptive statistics (n, mean, standard deviation, median, minimum, and maximum) will be provided for each time period and for changes from baseline for the following assessments: vital signs, Clinical Global Impression-Severity (CGI-S) questionnaire, Aberrant Behavioural Checklist (A-B-C), Autism Parenting Stress Index (APSI), Sleep disturbance scale for Children (SDSC) (parent or carer rated), Insomnia Severity Index (ISI)

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
15/04/2021
Actual
Date of last participant enrolment
Anticipated
31/12/2021
Actual
Date of last data collection
Anticipated
1/06/2022
Actual
Sample size
Target
150
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,WA,VIC
Recruitment hospital [1] 18504 0
Emerald Clinics Ltd - West Leederville - West Leederville
Recruitment postcode(s) [1] 32823 0
6007 - West Leederville

Funding & Sponsors
Funding source category [1] 307669 0
Commercial sector/Industry
Name [1] 307669 0
Zelira Therapeutics Ltd
Country [1] 307669 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Zelira Therapeutics Ltd
Address
Level 26, 140 St Georges Terrace
Perth WA 6000
Country
Australia
Secondary sponsor category [1] 308366 0
None
Name [1] 308366 0
Address [1] 308366 0
Country [1] 308366 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 307708 0
NIIM HREC
Ethics committee address [1] 307708 0
National Institute of Integrative Medicine
11-23 Burwood Road, Hawthorn, Melbourne
Victoria 3122 AUSTRALIA
Ethics committee country [1] 307708 0
Australia
Date submitted for ethics approval [1] 307708 0
27/10/2020
Approval date [1] 307708 0
21/01/2021
Ethics approval number [1] 307708 0
0080E_2020

Summary
Brief summary
This is an open label, observational study enrolling people (including children and adolescents (from 6 to 18 years) and adults) with ASD presenting with behavioural symptoms (including behaviour (aggressiveness, hyperactivity, irritability), social and emotional responsiveness, and communication skills) that receive medicinal cannabis as prescribed per standard practice. Patients will receive medicinal cannabis products HOPE 1 and/or HOPE 2 as per standard of care recommendations by their treating doctors. Patients will be assessed for their disease and safety according to standard practice. Each patient will be observed for a period of six months from the first visit.

Observation will only be on those patients that have been prescribed HOPE 1 and/or HOPE 2.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 108218 0
Dr Alistair Vickery
Address 108218 0
Emerald Clinics Ltd
Suite 9, Level 1
2 McCourt St
West Leederville WA 6007
Country 108218 0
Australia
Phone 108218 0
+61 08 6559 2829
Fax 108218 0
Email 108218 0
[email protected]
Contact person for public queries
Name 108219 0
Dr Alistair Vickery
Address 108219 0
Emerald Clinics Ltd
Suite 9, Level 1
2 McCourt St
West Leederville WA 6007
Country 108219 0
Australia
Phone 108219 0
+61 08 6559 2829
Fax 108219 0
Email 108219 0
[email protected]
Contact person for scientific queries
Name 108220 0
Dr Alistair Vickery
Address 108220 0
Emerald Clinics Ltd
Suite 9, Level 1
2 McCourt St
West Leederville WA 6007
Country 108220 0
Australia
Phone 108220 0
+61 08 6559 2829
Fax 108220 0
Email 108220 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Not applicable


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.