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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
Date last updated
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
This 18-week open-label study examines the effectiveness of Full-Spectrum Medicinal Cannabis Plant Extract containing 0.08% THC (NTI164) in treating Rett syndrome (RTT) in children and young people. The study aims to determine the impact of NTI164 on RTT symptoms over a 16-week treatment period.

Participants will start with a daily dose of 5 mg/kg of NTI164, which will be gradually increased over four weeks until they reach either the maximum tolerated dose or 20 mg/kg per day. They will then maintain this dose for eight weeks. Following this treatment phase, the dosage will be reduced by 5 mg/kg each week for four weeks until treatment concludes.

The effectiveness of the treatment will be assessed using tailored questionnaires that measure changes in the patients\' conditions. Additionally, full blood examinations will be conducted at multiple points throughout the study to monitor the effects of the treatment.
Trial website
https://clinicaltrials.gov/study/NCT06621043
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06621043