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Cannabidiol for severe behavioral manifestations in TSC, MPS III, and FXS894Rescue medicationThe study protocol does not specify any rescue medication to treat side effects of CBD treatment because, based on previous studies on the safety of CBD, these are usually mild at the doses used in this study. In case of SAEs, the patient will be withdrawn from the trial and appropriate treatment will be started.Outcome measures The primary outcome is a change on the irritability subscale score of the Aberrant Behavior Checklist (ABC-I) during active interventional periods compared to placebo periods. Secondary outcome measures include: y Total ABC;47y CGI;45y Syndrome-specific outcome measures, including the TSCspecific patient-reported outcome measure (TSC-PROM)48 and the Sanfilippo Behavior Rating Scale (SBRS);49y Pediatric Quality of Life Inventory (PedsQL);50y Anxiety, Depression, and Mood Scale (ADAMS);51y Social Communication Questionnaire (SCQ);52y Social Responsiveness Scale (SRS)-2 (when appropriate);53y Short Sensory Profile (SSP-NL);54y Parenting Stress Questionnaire (OBVL);55y Goal Attainment Scaling (GAS);56y Personal Questionnaire (PQ);57y Focal and generalized seizure frequency;y Adverse effects;y Hepatic enzyme levels.Baseline clinical characteristics, demographic information, medical history, results on CYP enzymes, and information regarding diagnosis will be collected and recorded in detail to enable better interpretation of results and explore factors associated with treatment response, because of the heterogeneity of the population and diverse neurobiological and behavioral phenotypes.44 Collecting data on CYP enzymes has already been Annelieke Muller sHL.indd 89 14-11-2023 09:07