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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
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
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
The Children’s Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
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
This is an 18-week open-label study to evaluate the efficacy of Full-Spectum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the severity of Rett syndrome (RTT) in children and young people.

The purpose of this study is to determine how effective NTI64 is in patients with RTT when treated for 16 weeks.

Participants will commence treatment with a daily dose of 5mg/kg of NTI164. This will gradually be increased 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 weeks (Treatment phase). At the end of the Treatment phase, participants will be gradually decreased by 5mg/kg for a period of 4 weeks until the end of their participation (Down-titration phase).

Efficacy will be measured and monitored by appropriate and releant questionnaires specific to measuring changes in patients.

Full blood examinations will be conducted at various points of the study.
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.