Page 62 - Demo

Chapter 360MethodsStudy designWe used the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) extension for N-of-1 trials (SPENT) checklist that is aligned with the CONSORT (consolidated reporting items for trials) extension for N-of-1 trials (CENT) for developing this N-of-1 protocol.29The study will consist of a series of N-of-1 trials followed by an optional open-label extension phase. Each trial is randomized, placebo-controlled, and double-blinded with multiple crossovers within a single patient. The trial consists of a baseline period, dose titration phase, and three cycles each consisting of one period of MPH treatment and one period of placebo treatment, both followed by a one-week washout period (Figure 1). Despite the fact that a one-day washout would suffice biologically, we chose oneweek washouts to account for prolonged psychological effects that may occur. The order of the treatment periods will be randomized. Thus, each N-of-1 trial will last 14 weeks with an additional follow-up measurement three months after completion of the N-of-1 trial.A: MethylphenidateB: PlaceboWashoutNo interventionFU: Follow-up measurementA Baseline periodCycle 1B BCycle 2A BCycle 3A0 6Number of weeks1 2 3 4 5 7 8 9 10 11 12 13FU27Dose titration14Dose 1Dose 2Dose 3Figure 1. Study design.Protocol development and patient engagementCollaboration with the Dutch SMS patient advocacy organization, caregivers of patients and clinical experts played a large role in defining knowledge and care gaps, prioritizing the treatment study, development of the current protocol and selecting outcome measures. We addressed specific difficulties for conducting this study, including concerns related to caregiver burden and patient burden of participation, and issues for recruitment and retention.Annelieke Muller sHL.indd 60 14-11-2023 09:07