Desmoid tumor - null regimens

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The purpose of this page is to provide references to "null therapy" such as placebo and observation. While not clinically relevant, these references provide further insight into the historical development of the treatment landscape. See the main desmoid tumor page for current regimens.

All lines of therapy



Study Years of enrollment Evidence Comparator Comparative Efficacy
Gounder et al. 2018 (Alliance A091105) 2014-2016 Phase 3 (C) Sorafenib Inferior PFS
Awaiting publication (DeFi) 2019-2022 Phase 3 (C) Nirogacestat TBD

No active antineoplastic treatment. Used as a comparator arm and here for reference purposes only.


  1. Alliance A091105: Gounder MM, Mahoney MR, Van Tine BA, Ravi V, Attia S, Deshpande HA, Gupta AA, Milhem MM, Conry RM, Movva S, Pishvaian MJ, Riedel RF, Sabagh T, Tap WD, Horvat N, Basch E, Schwartz LH, Maki RG, Agaram NP, Lefkowitz RA, Mazaheri Y, Yamashita R, Wright JJ, Dueck AC, Schwartz GK. Sorafenib for Advanced and Refractory Desmoid Tumors. N Engl J Med. 2018 Dec 20;379(25):2417-2428. link to original article contains dosing details in abstract link to PMC article PubMed NCT02066181
  2. DeFi: NCT03785964