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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06621043




Registration number
NCT06621043
Ethics application status
Date submitted
11/09/2024
Date registered
1/10/2024

Titles & IDs
Public title
Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)
Scientific title
Assessing the Safety and Efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) in the Treatment of Rett Syndrome (RTT)
Secondary ID [1] 0 0
NTIRTT1
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

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

Experimental: Active NTI164 Arm - Participants in this arm receive Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164), targeted at treating symptoms of Rett syndrome (RTT) in children and young people. The intervention starts with a daily initial dose of 5 mg/kg, which is increased gradually over a four-week up-titration phase to a maximum of 20 mg/kg per day, depending on individual tolerance. Participants then continue at their maximum tolerated dose for eight weeks during the treatment phase. The study concludes with a four-week down-titration phase, where the dosage is reduced by 5 mg/kg each week. The effectiveness of the treatment is monitored through specialized questionnaires and full blood examinations throughout the study duration.


Treatment: Drugs: NTI164
Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC, administered orally starting with a dose of 5 mg/kg, titrated up to 20 mg/kg based on tolerance, followed by a maintenance phase at the maximum tolerated dose, and concluding with a gradual dose reduction.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rett Syndrome: Symptom Index Score
Timepoint [1] 0 0
Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment
Secondary outcome [1] 0 0
Clinical Global Impression - Improvement (CGI-I)
Timepoint [1] 0 0
Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment
Secondary outcome [2] 0 0
Rett Syndrome Behaviour Questionnaire
Timepoint [2] 0 0
Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.
Secondary outcome [3] 0 0
RTT-Clinician Domain Specific Concerns - Visual Analog Scale
Timepoint [3] 0 0
Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.
Secondary outcome [4] 0 0
Communication and Symbolic Behaviour Scales Developmental Profile™ Infant-Toddler Checklist
Timepoint [4] 0 0
Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.
Secondary outcome [5] 0 0
Impact of Childhood Neurological Disability Scale
Timepoint [5] 0 0
Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.
Secondary outcome [6] 0 0
Rett Syndrome Caregiver Burden Inventory
Timepoint [6] 0 0
Baseline (pre-dose), 4, 12, 16 weeks post-commencement of treatment.
Secondary outcome [7] 0 0
The Overall Quality of Life Rating of the Impact of Childhood Neurological Disability Scale
Timepoint [7] 0 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
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



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

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/09/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
Accrual to date
Final
14
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
The Childrens Hospital at Westmead - Sydney
Recruitment postcode(s) [1] 0 0
2145 - Sydney

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

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

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

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06621043