Cannabis extract shows promise for rare genetic disorder in new trial

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Females aged 4-25 years of age 2. Weight ≥12 kg 3. Classical/typical RTT as confirmed with a documented pathogenic variant in the MECP2 gene 4. At least 6 months post-regression at screening (i.e. no loss or degradation in ambulation, hand function, speech, non-verbal communication, or social skills within 6 months of screening) 5. Rett Syndrome Clinical Severity Scale rating of 10-36 6. Clinical Global Impression - Severity of Illness score ≥4 7. Stable pattern of seizures or has had no seizures within 8 weeks of screening, as determined by the participant's primary physician 8. Other patient medications must be stable (i.e. no dose adjustments) for at least 8 weeks prior to screening, including steroids, anti-inflammatories, anxiolytics etc Exclusion Criteria: 1. 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 coeliac disease or inflammatory bowel disease), or major surgery planned 2. Known history or symptoms of long QT syndrome 3. QTcF interval \>450 milliseconds, history of risk factor for torsades de pointes or clinically significant QT prolongation deemed to increase risk 4. Currently receiving treatment with DAYBUE™ (Trofinetide) 5. Currently using other unregistered drugs for the treatment of Rett syndrome, such as Anavex® 6. Currently using or has used recreational or medicinal cannabis or 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 7. A known or suspected hypersensitivity to cannabinoids or any of the excipients 8. Moderate-severe impairment in hepatic function at screening, defined as serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 2 x upper limit of normal (ULN), or total bilirubin (TBL) \> 2 x ULN. This criterion can only be confirmed once laboratory results are available, participants enrolled into the trial who are later found to meet this criterion will be screen-failed. 9. Participant is enrolled in another clinical trial within 14 days of screening or becomes enrolled in another clinical trial throughout the duration of this study 10. Infection and/or antibiotic use in the 2 weeks prior to screening (participants can be recruited following 2 weeks without infection and/or antibiotic use)