Barbara Wendelberger

The primary objectives of a confirmatory clinical trial, namely a clinical trial conducted with the intention of changing clinical practice or obtaining regulatory approval, are typically to demonstrate and quantify the benefit of a treatment in a specific population of patients. Occasionally, accumulating evidence makes it clear that an ongoing clinical trial is unlikely to meet its original goals. When this occurs, the trial may be stopped early for futility.

Abstract Unlike familial Alzheimer’s disease, we have been unable to accurately predict symptom onset in presymptomatic familial frontotemporal dementia (f-FTD) mutation carriers, which is a major hurdle to designing disease prevention trials. We developed multimodal models for f-FTD disease progression and estimated clinical trial sample sizes in C9orf72, GRN and MAPT mutation carriers.