Sirolimus and Familial Adenomatous Polyposis (FAP)

Phase 2 Completed 4 enrolled Academic Medical Center - University of Amsterdam

NaSingle-groupOpen-labelTreatment

Summary

The aim of the study is to investigate the effect of sirolimus on the progression of intestinal adenomas in patients with FAP and to assess the safety of this treatment.

Timeline

Start: 2017-10-03
Primary completion: 2018-12-10
Completion: 2018-12-10

Outcome

Results reported · no primary-endpoint met/missed verdict (typical of a dose-finding / safety study)

paper Primary outcomes were safety and change in marked polyp size. PMID 33376109 ↗

Publications

Roos VH, Meijer BJ, Kallenberg FGJ, Bastiaansen BAJ, Koens L, Bemelman FJ, Bossuyt PMM, Heijmans J, van den Brink G, Dekker E. Sirolimus for the treatment of polyposis of the rectal remnant and ileal pouch in four patients with familial adenomatous polyposis: a pilot study. BMJ Open Gastroenterol. 2020 Dec;7(1):e000497. doi: 10.1136/bmjgast-2020-000497.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Sirolimus	Small molecule	2 mg	Oral