Page 67 - Demo

Methylphenidate for ADHD in Smith-Magenis syndrome653completing the three cycles or in case of serious adverse events (SAEs). Investigators involved in data analysis will remain blinded until the end of the follow-up period.Multi-site training planA pre-study training meeting will be planned to train clinical investigators and clinical evaluators on study procedures and GAS with a secondary goal to promote reliability of GAS. All clinical and research staff that is involved in either identification or assessment of goals by GAS will be trained by a GAS expert to promote data quality.Interventions and dosing scheduleOne dose titration kit and a trial kit including MPH (regular tablet) and placebo will be developed and distributed by the Amsterdam UMC hospital pharmacist.Dose titration phaseThe MPH dosage will be titrated to achieve the maximum dosage with minimal side effects determined by the psychiatrist or ID physician. Titration dosage will be blinded to the participants and caregivers and comprise two days each of three escalating doses in steps of 2.5 mg of MPH with a total of six days followed by a washout period of at least one week. The individually determined starting dose for the dose titration phase will be based on age and body weight. During the dose titration phase, participants will daily fill out the checklist of side effects of MPH.40 MPH effectiveness will explicitly not be examined during the titration phase to prevent high dropout rates when participants might get prematurely convinced about the effectiveness.TrialDuring the N-of-1 trial, MPH dosage as determined by titration phase or placebo will be administered by caregivers twice daily during breakfast and during lunch (around 7.30 am and 12.30 pm). During washout periods, the placebo will be administered.Annelieke Muller sHL.indd 65 14-11-2023 09:07