ACTRN12623000563662
Recruiting
Phase 1
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 Group0 sites14 target enrollmentMay 25, 2023
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Rett Syndrome
- Sponsor
- Fenix Innovation Group
- Enrollment
- 14
- Status
- Recruiting
- Last Updated
- 2 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
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.
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.
Outcomes
Primary Outcomes
Not specified
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