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Brief Title: Study of Everolimus in the Treatment of Advanced Malignancies in Patients With Peutz-Jeghers Syndrome
Official Title: Pilot Study of Everolimus in the Treatment of Neoplasms in Patients With Peutz-Jeghers Syndrome
Study ID: NCT01178151
Brief 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.
Detailed Description:
Minimum Age: 18 Years
Eligible Ages: ADULT, OLDER_ADULT
Sex: ALL
Healthy Volunteers: No
Academic Medical Center, Amsterdam, , Netherlands
Erasmus Medical Center, Rotterdam, , Netherlands
Name: Heinz-Josef Klumpen, MD
Affiliation: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Role: PRINCIPAL_INVESTIGATOR