On December 18th 2024 Britt Dhaenens defended her thesis Innovative designs to improve trials in NF.
NF and SWN predispose to a specific spectrum of associated tumours and other disease manifestations. Given the ineffectiveness of surgery and radiation therapy, there is great need for drug treatments for NF-associated tumours. However, there has been a limited translation from drug candidates to drug approval. This can be explained by a number of challenges in clinical trials in NF, including small available sample sizes, a lack of natural history data, variable treatment needs between patients, and a lack of validated outcomes, in particular patient-reported outcome measures. The aim of this thesis is to contribute to the improvement of clinical trials for NF, by identifying and addressing specific challenges in trials for these conditions.
Read the thesis here