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
Outcome
Met primary endpoint
paper A binomial exact test showed that TTP2 was significantly higher than TTP1 (GMI 80.0%; 95% CI, 60.0-100; p = 0.0059) PMID 41903360 ↗
paper indicating that the study's primary objective was met. PMID 41903360 ↗
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
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Toremifene | Small molecule | 60 mg | Oral |
| Subject | Toremifene | Small molecule | 180 mg | Oral |