Page 87 - Demo

Cannabidiol for severe behavioral manifestations in TSC, MPS III, and FXS854N-of-1 trial consists of two cycles, each consisting of one period of CBD treatment (A; six weeks), one period of placebo treatment (B; six weeks), run-in periods (three weeks), taper periods (two weeks), and washouts following A and B (one week) (Figure 1). The total duration of each trial will last around one year. The optional open-label extension phase will be a further twelve months. BlockrandomizationA: CBD Contact moment (secondary outcome measures)B: PlaceboWashout Taper * Primary outcome measureRun-in No interventionBaselineCycle 1BCycle 2weeksDose titration-8 -6 -3 -1 0 3 9 11 12 15 21 23 24 27 33 35 36 39 45A A B* * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * * *VariableDeblindingFUOptional open-label extension phase (12 months)*Figure 1. Study design of the N-of-trial.CBD, cannabidiol; FU, follow-up. Study settingPrior to the start of the trial, all clinical measures and questionnaires will be completed in the Amsterdam UMC, location AMC. The remaining questionnaires will be completed and study medication will be taken at the participants’ natural setting.RecruitmentParticipants will be recruited through the outpatient clinics for TSC and FXS at ‘s Heeren Loo, the UMC Utrecht and the Erasmus MC, and through patient organizations and the MPS III expert center at the Amsterdam UMC.Study populationThe study population consists of children and adults with TSC and FXS, and children with MPS III, all suffering from severe behavioral manifestations. We aim to conduct a patient-centered trial, allowing for a natural setting and flexibility, including continuation of concurrent therapies. Annelieke Muller sHL.indd 85 14-11-2023 09:07