Pilot Study of mTOR Inhibitor Therapy in Peutz-Jeghers Syndrome (NCT00811590) | Clinical Trial Compass
TerminatedPhase 2
Pilot Study of mTOR Inhibitor Therapy in Peutz-Jeghers Syndrome
Stopped: The study was ended early due to low enrollment.
United States3 participantsStarted 2008-11
Plain-language summary
Pilot study, Open-label, Phase II study of Everolimus.
Objective:
To determine if Everolimus can diminish large gastrointestinal polyps in patients with Peutz-Jeghers Syndrome.
Methodology:
Polyp size and number will be compared to baseline by FDG-PET and CT and 12 months after treatment with Everolimus. Since this is a pilot study, the polyps prior to treatment will serve as the controls.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Patients who are 18 years or older with a clinical or genetic diagnosis of Peutz-Jeghers Syndrome.
✓. Patient has one or more intestinal polyps ≥ 5mm in maximum diameter by contrast enhanced CT scan that is not clinically indicated for removal or is beyond the reach of a push endoscope.
✓. Minimum of two weeks since any major surgery.
✓. Patient has had colonoscopy within the past 24 months and did not have high-grade dysplasia or colorectal cancers.
✓. WHO performance status £ 2
✓. Adequate bone marrow function as shown by: ANC ≥ 1.5 x 109/L, Platelets ≥ 100 x 109/L, Hgb \> 9 g/dL
✓. Adequate liver function as shown by: serum bilirubin ≤ 1.5 x upper limit of normal (ULN), and serum transaminases activity ≤ 2.5 x ULN.
✓. Patients must be able to provide written informed consent.
Exclusion criteria
✕. Prior treatment with any investigational drug within the preceding 4 weeks
✕. Chronic treatment with systemic steroids or another immunosuppressive agent
✕. Patients should not receive immunization with attenuated live vaccines during study period or within one week of study entry
✕. Uncontrolled brain or leptomeningeal metastases, including patients who continue to require glucocorticoids for brain or leptomeningeal metastases
✕. Other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell carcinomas of the skin.
✕. Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
✕. Unstable angina pectoris, symptomatic congestive heart failure, myocardial infarction ≤ 6 months prior to first study treatment, serious uncontrolled cardiac arrhythmia