ANHL2121: Phase 2 Study of Tovorafenib (DAY101) in Relapsed and Refractory Langerhans Cell Histiocytosis
What is the goal of the study?
Recent discoveries support reclassification of LCH as a tissue-infiltrating, clonal neoplasm derived from myeloid dendritic cells showing a Langerhans cell phenotype. Somatic BRAF-V600E and MAP2K1 mutations have been identified in ~85% of cases of LCH, and pathologic MAPK pathway activation is considered to be universal in LCH. This trial is an open-label, multi-center Phase 2 study to evaluate the safety and efficacy of the pan-RAF inhibitor DAY101 in patients with relapsed/refractory LCH with pathologic somatic mutations in genes encoding tyrosine kinase receptors, RAS or RAF. DAY101 will be given orally at the recommended phase 2 dose (RP2D). The primary end point will be the overall response rate. Event-free, progression-free, and overall survival will be assessed as secondary end points. Tumor biopsies and serial blood specimens will be collected for correlative biology.
Who can participate in the study?
Please contact the study team listed below to learn more.