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 个研究点目标入组 14 人开始时间: 2023年5月25日最近更新: 2023年11月6日

适应症

试验速览

暂无简介。

•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.

•- 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.

发起方

Fenix Innovation Group