Status:
TERMINATED
Pilot Study of mTOR Inhibitor Therapy in Peutz-Jeghers Syndrome
Lead Sponsor:
University of Utah
Collaborating Sponsors:
Novartis
Conditions:
Peutz-Jeghers Syndrome
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief 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 ...
Detailed Description
Peutz-Jeghers Syndrome is a hereditary polyposis condition in which hamartomatous tumors develop in many tissues of the body. These tumors are benign but frequently cause gastrointestinal obstruction ...
Eligibility Criteria
Inclusion
- Yes/No (Response of "no" = patient ineligible)
- 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
- Yes/No (Response of "yes" = patient ineligible)
- 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
- Severely impaired lung function
- Uncontrolled diabetes as defined by fasting serum glucose \>1.5x ULN
- Any active (acute or chronic) or uncontrolled infection/ disorders.
- Nonmalignant medical illnesses that are uncontrolled or whose control may be jeopardized by the treatment with the study therapy
- Liver disease such as cirrhosis, or severe hepatic impairment (Child-Pugh class C)
- A known history of HIV seropositivity
- Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of everolimus (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection)
- Patients with an active, bleeding diathesis or on oral anti-vitamin K medication
- Women who are pregnant or breast feeding, or women/men able to conceive and unwilling to practice an effective method of birth control throughout the trial and for 8 weeks after the last dose of study drug. (Women of childbearing potential must have a negative pregnancy test). Oral, implantable, or injectable contraceptives may be affected by cytochrome P450 interactions, and are therefore not considered effective for this study.
- Male patients whose sexual partner(s) are WOCBP who are not willing to use adequate contraception during the study and for 9 weeks after the end of treatment.
- Patients who have received treatment with an mTor inhibitor in the past 6 months.
- Patients with a known hypersensitivity to RAD001 (everolimus) or other rapamycins (sirolimus, temsirolimus) or to its excipients
- Patients who can not undergo FDG-PET are eligible to participate in this study for the purpose of the primary endpoint. Patient with the following will be excluded from FDG-PET piece of the study.
- Patients cannot have a serum glucose level greater than 200 mg/dl for FDG-PET imaging
- Patients who are too claustrophobic to undergo FDG-PET imaging
- Patients who will require conscious sedation to undergo FDG-PET imaging.
Key Trial Info
Start Date :
November 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2011
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00811590
Start Date
November 1 2008
End Date
March 1 2011
Last Update
September 19 2024
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Huntsman Cancer Institute
Salt Lake City, Utah, United States, 84112