The abstract is read at the publisher; the summary is JClub's.
In adults with histologically confirmed desmoid tumors, varegacestat produced the largest week-16 tumor volume reduction with 1.2 mg once daily (-51.91%) and a 35.7% confirmed ORR.
- Why it matters: Desmoid tumors are rare and often locally aggressive, yet effective treatments remain limited, creating a need to define safer, more effective dosing strategies.
- What they did: In the open-label, randomized RINGSIDE phase II trial, 42 adults with progressing desmoid tumors received 1.2-mg once daily, 2-mg intermittent, or 4-mg intermittent dosing; safety was primary and tumor volume change at week 16 was assessed by blinded independent central review.
- The result: Treatment-related adverse events occurred in 41/42 (97.6%) participants, with grade 3 AEs in 9 (21.4%) and no serious AEs or grade 4/5 events; in the 1.2-mg open-label extension, median best tumor volume change was -85.88% and confirmed ORR rose to 57.1%.
The findingWhy it mattersWhat they didThe result
AI summary of the abstract · may contain errors · not medical advice · read the paper.