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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)
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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.
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Secondary ID [1]
309657
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NTIRTT1
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome
329995
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Condition category
Condition code
Neurological
326905
326905
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0
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Neurodegenerative diseases
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
326091
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Treatment: Drugs
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Comparator / control treatment
No control group
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Control group
Uncontrolled
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Outcomes
Primary outcome [1]
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Change in Clinical Global Impression
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Assessment method [1]
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Timepoint [1]
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [1]
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Rett Syndrome: Symptom Index Score (RTT-SIS)
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Assessment method [1]
421933
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Timepoint [1]
421933
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [2]
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Rett Syndrome Behaviour Questionnaire (RSBQ)
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Assessment method [2]
422155
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Timepoint [2]
422155
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [3]
422156
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RTT- Clinician Domain Specific Concerns – Visual Analog Scale (RTT-DSC-VAS)
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Assessment method [3]
422156
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Timepoint [3]
422156
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [4]
422157
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Communication and Symbolic Behaviour Scales Developmental Profile™ Infant-Toddler Checklist (CSBS-DP-IT Social)
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Assessment method [4]
422157
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Timepoint [4]
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [5]
422158
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Impact of Childhood Neurological Disability Scale (ICND)
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Assessment method [5]
422158
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Timepoint [5]
422158
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [6]
422159
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RTT Caregiver Burden Inventory (RTT-CBI)
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Assessment method [6]
422159
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Timepoint [6]
422159
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [7]
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Overall Quality of Life Rating of the Impact of Childhood Neurological Disability Scale (ICND-QoL)
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Assessment method [7]
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Timepoint [7]
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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Secondary outcome [8]
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full blood examination, liver and renal function to monitor safety.
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Assessment method [8]
422161
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Timepoint [8]
422161
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Baseline (pre-dose), 4, 12 & 16 weeks post-commencement of treatment.
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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.
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Minimum age
5
Years
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Maximum age
20
Years
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Sex
Females
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
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Who is / are masked / blinded?
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Intervention assignment
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Other design features
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Phase
Phase 1 / Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Date of first participant enrolment
Anticipated
17/07/2023
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Actual
20/09/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
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Sample size
Target
14
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Accrual to date
1
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Final
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
24719
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The Children's Hospital at Westmead - Westmead
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Recruitment postcode(s) [1]
40338
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2145 - Westmead
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Funding & Sponsors
Funding source category [1]
313848
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Commercial sector/Industry
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Name [1]
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Fenix Innovation Group
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Address [1]
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5A Hartnett Close, Mulgrave VIC Australia 3170
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Country [1]
313848
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Australia
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Funding source category [2]
313849
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Commercial sector/Industry
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Name [2]
313849
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Neurotech International Limited
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Address [2]
313849
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Suite 5 CPC
145 Stirling Highway Nedlands WA 6009
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Country [2]
313849
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Australia
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Primary sponsor type
Commercial sector/Industry
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Name
Fenix Innovation Group
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Address
5A Hartnett Close, Mulgrave VIC 3170
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Country
Australia
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Secondary sponsor category [1]
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None
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Name [1]
315680
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Address [1]
315680
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Country [1]
315680
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Ethics approval
Ethics application status
Approved
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Ethics committee name [1]
312997
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The Sydney Children's Hospitals Network (SCHN) Human Research Ethics Committee (HREC)
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Ethics committee address [1]
312997
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The Children’s Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
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Ethics committee country [1]
312997
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Australia
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Date submitted for ethics approval [1]
312997
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17/05/2023
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Approval date [1]
312997
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10/07/2023
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Ethics approval number [1]
312997
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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.
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Trial website
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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A/Prof Carolyn Ellaway
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Address
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The Children's Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
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Country
126630
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Australia
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Phone
126630
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+610298453654
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Fax
126630
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Email
126630
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[email protected]
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Contact person for public queries
Name
126631
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A/Prof Carolyn Ellaway
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Address
126631
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The Children's Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
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Country
126631
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Australia
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Phone
126631
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+61029845 3654
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Fax
126631
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Email
126631
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[email protected]
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Contact person for scientific queries
Name
126632
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A/Prof Carolyn Ellaway
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Address
126632
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The Children's Hospital at Westmead
Corner Hawkesbury Road and Hainsworth Street
Westmead NSW 2145
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Country
126632
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Australia
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Phone
126632
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+610298453654
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Fax
126632
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Email
126632
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[email protected]
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Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
IPD will not be shared as per commercial in confidence restrictions.
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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.
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