Toremifene in Desmoid Tumor: Prospective Clinical Trial and Identification of Potential Molecular Targets

Summary

This is a prospective study evaluating the activity and the safety of toremifene in patients with primary or recurrent sporadic DTs. Patients will be enrolled after the histological confirmation of DTs on biopsy Patients will start at 60 mg daily and dose-escalate to 180 mg upon progression. Disease assessment will be performed by contrast-enhanced MRI or CT scan, pain evaluation by a visual analog scale (VAS) every 3 months for the first and second year, twice yearly thereafter. Response will be evaluated either by RECIST and/or symptomatic relief.

Timeline

Start

2012-11

Primary completion

2015-06

Completion

2015-12

Publications

• Colombo C, Tine G, Palassini E, Belfiore A, Marrari A, Collini P, Fiore M, Radaelli S, Perrone F, Miceli A, Gronchi A. Toremifene in desmoid fibromatosis: Prospective phase 2 study of two dose levels. Eur J Cancer. 2026 May 15;239:116673. doi: 10.1016/j.ejca.2026.116673. Epub 2026 Mar 20.
• Fiore M, Colombo C, Radaelli S, Callegaro D, Palassini E, Barisella M, Morosi C, Baldi GG, Stacchiotti S, Casali PG, Gronchi A. Hormonal manipulation with toremifene in sporadic desmoid-type fibromatosis. Eur J Cancer. 2015 Dec;51(18):2800-7. doi: 10.1016/j.ejca.2015.08.026. Epub 2015 Nov 18.

Drugs

Indications