Bernd Kasper

RINGSIDE Phase II Study of Varegacestat for Treatment of Desmoid Tumors: A Randomized Dose-Finding Clinical Trial

PURPOSE Desmoid tumors (DTs) are rare, often locally aggressive, connective tissue neoplasms with limited effective treatment options. We evaluated the safety and efficacy of varegacestat, an oral gamma-secretase inhibitor. METHODS RINGSIDE phase II was an open-label, randomized,...

Management of Soft Tissue and Visceral Leiomyosarcomas

Importance Leiomyosarcoma is a rare and heterogeneous malignant mesenchymal neoplasm associated with substantial morbidity and mortality. Given recent advances in biologic understanding and the complexity of leiomyosarcoma, a consensus-driven approach is needed to harmonize manag...

Patient-centred and fit-for-purpose clinical trials in rare cancers: lessons from sarcomas for operationalising 2024 WHO Guidance and ICH E6(R3)

Rare cancers account for approximately one quarter of cancer diagnoses in Europe, yet prevailing clinical trial governance frameworks were developed for common malignancies and conventional randomised designs. The 2024 WHO Guidance for best practices for clinical trials and ICH E...