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The first randomised trial to show a drug controls desmoid tumours, with better pain and function.
DeFi, trial NCT03785964 sponsored by SpringWorks and published in the New England Journal of Medicine in 2023, was the first randomised trial to show that a drug controls desmoid tumours, with better pain and physical function. It randomised 142 patients with progressing desmoid tumours to the gamma-secretase inhibitor nirogacestat or placebo, met its primary progression-free survival endpoint with a very large effect, produced responses in 41 percent against 8 percent including some complete responses, and led to approval in November 2023. OnCo links it to desmoid tumour and sarcomas, nirogacestat, the Wnt and beta-catenin pathway, the bottleneck of rare cancers without markets and the idea of intermittent or stop-and-restart dosing. Whether treatment can be paused once tumours are controlled is the open question.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
142 enrolled.
Shares Gamma-secretase inhibitors, Nirogacestat, Desmoid tumour, Small-molecule kinase inhibitors.
Shares Gamma-secretase inhibitors, Nirogacestat, Small-molecule kinase inhibitors.
Shares Gamma-secretase inhibitors, Nirogacestat, Desmoid tumour.
Shares Gamma-secretase inhibitors, Nirogacestat, Small-molecule kinase inhibitors.
Shares Gamma-secretase inhibitors, Nirogacestat, Small-molecule kinase inhibitors.
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).
Shares Rare and paediatric cancers without markets, Sarcomas (soft tissue, bone, GIST).