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Chapter 4100and design used, an individual may have been on a placebo for the entire trial. Moreover, data from the current series of N-of-1 trials will be pooled to obtain a population treatment effect estimate.In conclusion, we consider that the N-of-1 trial design is excellent to study pharmacological treatments of disease manifestations in rare populations. The current study will provide crucial information about the efficacy of CBD for severe behavioral manifestations in these complex and vulnerable patient populations. AcknowledgementsThe authors would like to thank the Dutch patient advocacy organizations (Stichting Tubereuze Sclerosis Nederland, Fragiele X Vereniging Nederland) for their feedback on the study protocol. The Amsterdam UMC and Erasmus MC are member of European Reference Network (ERN) ITHACA. The Amsterdam UMC is member of MetabERN. The UMC Utrecht is member of ERN EpiCare. FundingThe trial is financially sponsored by a grant from GWResearch/Jazz pharmaceuticals and the Dutch TSC foundation. The open-label extension is also funded by Jazz Pharmaceuticals. Competing interestsThe authors declare that they have no competing interest.Authors’ contributionsAM and AvE proposed the study. AM and BdH initiated the design and wrote the study protocol. PvdV designed the statistical analysis and performed the analyses. KR provided methodological expertise. LG conducted EEG measurements. HB supervised the study design and conducted EEG measurements. CvK, FJ, MdW, LtH, AR, BD, FW, and EB supervised the study design. MB and AvE supervised the study design and wrote the study protocol. All authors provided critical feedback, read, and approved the final manuscript.Annelieke Muller sHL.indd 100 14-11-2023 09:07