Page 88 - Demo

Chapter 486Inclusion criteria include:y Minimum age of 6 years old.y Clinically and/or genetically definite diagnosis of TSC, MPS III or FXS (modified Gomez Criteria, clinical criteria, positive genetic test, or enzyme deficiency).y Suffering from severe behavioral manifestation with a minimum score of 4 on the Clinical Global Impression - severity (CGI-S) scale.45y Stable dose of all psychopharmacological medications or interventions for one month prior to screening and willingness of the participant and legal representatives to maintain the current medication regimen throughout the trial.y Presence of a consistently available patient caregiver for proxy reports.Exclusion criteria include:y Any known or suspected hypersensitivity to cannabinoids or any of the excipients of the Investigational Medicinal Product (IMP), such as sesame oil.y History of recreational or medicinal cannabis, or cannabinoidbased medications, with three months prior to screening and the patient is unwilling to abstain for the duration of the study.y History or current evidence of significantly impaired liver function, defined as 1) Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 5 x upper limit of normal (ULN); 2) ALT or AST > 3 x ULN with concomitant total bilirubin > 2.0 x ULN; or 3) ALT or AST ≥ 3 x ULN with the appearance of fatigue, nausea, vomiting, right upper quadrant pain or tenderness, fever, rash, and/or eosinophilia. y Pregnancy or breastfeeding. Females of childbearing potential must be willing to use an effective method of contraception from the time of consent until six weeks after treatment discontinuation and inform the trial if pregnancy occurs.y Glaucoma.y History of general anesthesia in the four weeks prior to enrolment.Annelieke Muller sHL.indd 86 14-11-2023 09:07