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Trial registered on ANZCTR


Registration number
ACTRN12623000563662
Ethics application status
Approved
Date submitted
14/05/2023
Date registered
25/05/2023
Date last updated
27/10/2023
Date data sharing statement initially provided
25/05/2023
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase I/II study to assess the safety and efficacy of full-spectrum medicinal cannabis plant extract 0.08% THC (NTI164) in the treatment of Rett syndrome (RTT)
Scientific title
A Phase I/II study to assess the safety and efficacy of full-spectrum medicinal cannabis plant extract 0.08% THC (NTI164) in the treatment of Rett syndrome (RTT) in female patients aged 5 to 16 years.
Secondary ID [1] 309657 0
NTIRTT1
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome 329995 0
Condition category
Condition code
Neurological 326905 326905 0 0
Neurodegenerative diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
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 an 8-week period.
• 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.

Adherence to intervention will be monitored by drug product return accountability, completion of online drug administration forms and study-specific questionnaires.
Intervention code [1] 326091 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 334746 0
Change in Clinical Global Impression
Timepoint [1] 334746 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [1] 421933 0
Rett Syndrome: Symptom Index Score (RTT-SIS)
Timepoint [1] 421933 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [2] 422155 0
Rett Syndrome Behaviour Questionnaire (RSBQ)
Timepoint [2] 422155 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [3] 422156 0
RTT- Clinician Domain Specific Concerns – Visual Analog Scale (RTT-DSC-VAS)
Timepoint [3] 422156 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [4] 422157 0
Communication and Symbolic Behaviour Scales Developmental Profile™ Infant-Toddler Checklist (CSBS-DP-IT Social)
Timepoint [4] 422157 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [5] 422158 0
Impact of Childhood Neurological Disability Scale (ICND)
Timepoint [5] 422158 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [6] 422159 0
RTT Caregiver Burden Inventory (RTT-CBI)
Timepoint [6] 422159 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [7] 422160 0
Overall Quality of Life Rating of the Impact of Childhood Neurological Disability Scale (ICND-QoL)
Timepoint [7] 422160 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
Secondary outcome [8] 422161 0
full blood examination, liver and renal function to monitor safety.
Timepoint [8] 422161 0
Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.

Eligibility
Key inclusion criteria
- Girls and women, aged 5-20 years
- Weight greater than or equal to 12kg
- Classical/typical RTT
- Documented disease-causing mutation in MECP2 gene
- At least 6 months post regression at screening (ie. no loss or degradation in ambulation, hand function, speech, nonverbal communicative or social skills within 6 months of screening)
Rett Syndrome Clinical Severity Scale rating of 10-36
- CGI score of 4 or higher.
- Stable pattern of seizures, or has had no seizures, within 8 weeks of screening.
Minimum age
5 Years
Maximum age
20 Years
Sex
Females
Can healthy volunteers participate?
No
Key exclusion criteria
- Current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, type 1 diabetes, or uncontrolled type 2 diabetes), renal, hepatic, respiratory, or gastrointestinal disease (such as celiac disease or inflammatory bowel disease), or major surgery planned during the study.
- Known history or symptoms of long QT syndrome.
- QTcF interval >450 ms, history of risk factor for torsades de pointes or clinically significant QT prolongation deemed to increase risk.
- Treatment with insulin, IGF-1, or growth hormone within 12 weeks of baseline.
- Currently receiving treatment with DAYBUETM (trofinetide).
- Currently using other unregistered drugs for the treatment of Rett syndrome such as Anavex.
- 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Phase 1 / Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
17/07/2023
Actual
20/09/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
14
Accrual to date
1
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 24719 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 40338 0
2145 - Westmead

Funding & Sponsors
Funding source category [1] 313848 0
Commercial sector/Industry
Name [1] 313848 0
Fenix Innovation Group
Country [1] 313848 0
Australia
Funding source category [2] 313849 0
Commercial sector/Industry
Name [2] 313849 0
Neurotech International Limited
Country [2] 313849 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Fenix Innovation Group
Address
5A Hartnett Close, Mulgrave VIC 3170
Country
Australia
Secondary sponsor category [1] 315680 0
None
Name [1] 315680 0
Address [1] 315680 0
Country [1] 315680 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 312997 0
The Sydney Children's Hospitals Network (SCHN) Human Research Ethics Committee (HREC)
Ethics committee address [1] 312997 0
Ethics committee country [1] 312997 0
Australia
Date submitted for ethics approval [1] 312997 0
17/05/2023
Approval date [1] 312997 0
10/07/2023
Ethics approval number [1] 312997 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 126630 0
A/Prof Carolyn Ellaway
Address 126630 0
The Children's Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
Country 126630 0
Australia
Phone 126630 0
+610298453654
Fax 126630 0
Email 126630 0
carolyn.ellaway@health.nsw.gov.au
Contact person for public queries
Name 126631 0
Carolyn Ellaway
Address 126631 0
The Children's Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
Country 126631 0
Australia
Phone 126631 0
+61029845 3654
Fax 126631 0
Email 126631 0
carolyn.ellaway@health.nsw.gov.au
Contact person for scientific queries
Name 126632 0
Carolyn Ellaway
Address 126632 0
The Children's Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
Country 126632 0
Australia
Phone 126632 0
+610298453654
Fax 126632 0
Email 126632 0
carolyn.ellaway@health.nsw.gov.au

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
IPD will not be shared as per commercial in confidence restrictions.


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.