N-of-1 trials are randomised, controlled, multiple crossover trials in single patients, in which data are contributed over several treatment cycles.
Following completion of the multiple crossover part of the trial, the results of each n-of-1 trial will be analysed by the researchers and a report will be drafted for each patient individually (weeks 7 and 8).
Specific criteria for withdrawal of individual participants consist of: The occurrence of a SAE or SUSAR; A myasthenic crisis or worsening of symptoms requiring any treatment other than the trial medication; A need to undergo thymectomy before the end of a cycle in the multiple crossover phase of the trial.
The occurrence of a SAE or SUSAR; A myasthenic crisis or worsening of symptoms requiring any treatment other than the trial medication; A need to undergo thymectomy before the end of a cycle in the multiple crossover phase of the trial.
Unblinding will occur when a participant has completed the multiple crossover phase of the trial or in the case of a serious adverse event (SAE) which cannot be treated without knowing which treatment the patient was receiving (see also 'Safety Evaluation' section).
23 25 Given the multiple crossover character of n-of-1 trials (ie, each patient acts as their own control and contributes multiple cycles of data), the number of patients needed in n-of-1 trials (ie, sample size) to estimate the population treatment effect is much reduced compared with group-RCTs 26 and thus makes this type of trial very suitable to conduct research in small patient populations.
This study was a single-centre (Department of Internal Medicine, Division of Endocrinology and Metabolism, Medical University of Graz, Austria), randomised, multiple-dose, double-blind, two-period, crossover trial conducted in subjects with type 1 diabetes (Clinical trials.gov number: NCT00964418).
Patients and physicians will be blinded to the treatment sequence during the multiple crossover phase of the n-of-1 trial.
Since AEs are strictly monitored as outlined earlier and the duration of blinding is only short (6 weeks during the multiple crossover phase of the n-of-1 trials), no additional data monitoring committee will be implemented.
A descriptive report will list the number of patients who experienced adverse effects during the placebo and ephedrine periods of the multiple crossover phase of the n-of-1 trials, as well as the type of adverse effect.
