Study of Everolimus in the Treatment of Advanced Malignancies in Patients With Peutz-Jeghers Syndrome

Phase 2 Withdrawn Academic Medical Center - University of Amsterdam Erasmus Medical Center · collab

NaSingle-groupOpen-labelTreatment

Summary

In this pilot study the investigators will treat all patients known with Peutz-Jeghers syndrome (PJS) who are diagnosed with advanced malignancies with everolimus 10mg daily until disease progression. Most patients with PJS have an inherited LKB1 mutation leading to aberrant m-TOR activity. Their risk to develop malignancies or intestinal polyps is probably related to this constitutive mTOR signaling. The hypothesis is that mTOR inhibition is an effective anticancer treatment in PJS patients with advanced malignancies.

Timeline

Start: 2010-10
Primary completion: 2015-04
Completion: 2015-04

Outcome

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

Stopped: “No patients”

paper Due to accrual problems, no conclusions can be drawn about the value of everolimus in PJS treatment PMID 29371475 ↗

Publications

de Brabander J, Eskens FALM, Korsse SE, Dekker E, Dewint P, van Leerdam ME, van Eeden S, Klumpen HJ. Chemoprevention in Patients with Peutz-Jeghers Syndrome: Lessons Learned. Oncologist. 2018 Apr;23(4):399-e33. doi: 10.1634/theoncologist.2017-0682. Epub 2018 Jan 25.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Everolimus	Small molecule	10 mg	Oral