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)
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.
Fenix Innovation Group
14 participants
Sep 20, 2023
Interventional
Conditions
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.
Eligibility
Inclusion Criteria8
- 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.
Exclusion Criteria8
- 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.
Interested in this trial?
Get notified about updates and connect with the research team.
Interventions
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.
Locations(1)
View Full Details on ANZCTR
For the most up-to-date information, visit the official listing.
ACTRN12623000563662