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Sponsors and Collaborators: |
University of Utah Novartis |
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Information provided by: | University of Utah |
ClinicalTrials.gov Identifier: | NCT00811590 |
Pilot study, Open-label, Phase II study of RAD001.
Objective:
To determine if RAD001 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 RAD001. Since this is a pilot study, the polyps prior to treatment will serve as the controls.
Condition | Intervention | Phase |
---|---|---|
Peutz-Jeghers Syndrome |
Drug: RAD001 |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Active Control, Single Group Assignment |
Official Title: | Pilot Study of mTOR Inhibitor Therapy for Treatment of Intestinal Polyps in Peutz-Jeghers Syndrome |
Estimated Enrollment: | 10 |
Study Start Date: | November 2008 |
Estimated Study Completion Date: | December 2011 |
Estimated Primary Completion Date: | December 2011 (Final data collection date for primary outcome measure) |
RAD001 (everolimus) is a novel derivative of rapamycin. RAD001 has been in clinical development since 1996 as an immunosuppressant in solid organ transplantation. Since 2003, RAD001 is approved in Europe (trade name: Certican) via the Mutual Recognition Procedure (MRP) for the prevention of organ rejection in patients with renal and cardiac transplantation. Certican is also approved in Australia, South Africa, the Middle East, Central and South America, the Caribbean and some Asian countries.
RAD001 is being investigated as an anticancer agent based on its potential to act
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 and bleeding beginning in the 2nd-3rd decades of life necessitating surgical intervention. Unfortunately, a recent study showed that the lifetime risk of cancers that arise in Peutz-Jeghers Syndrome is 85% by age 70 years and is 60% by age 60 years (Hearle et al., 2006).
A working definition of PJS has been suggested by Giardiello et al ,1987(www.genetests.com):
For individuals with a histopathologically confirmed hamartoma, a definite diagnosis of PJS requires two of the following three findings:
Recently, rapamycin (Rapamune, Wyeth), an FDA-approved drug for use in orthotopic transplant recipients, was successfully used in an off-label study of 5 individuals with a related condition called tuberous sclerosis in which the patients had subependymal giant cell astrocytomas that caused significant and insidious neurological problems such as hydrocephalus and seizures (Franz, et al. 2006). All astrocytoma lesions exhibited regression with treatment of oral rapamycin and in one case, necrosis. Treatment was well tolerated and may offer an alternative to operative therapy in tuberous sclerosis. Tuberous sclerosis is caused by germline mutations in the tuberous sclerosis 1 or 2 genes. These genes encode proteins that function downstream of STK11, the gene that is mutated in Peutz-Jeghers Syndrome. Mutations of STK11 or TSC1/2 leads to activation of mTOR (mammalian target of rapamycin). Dysregulation of mTOR has been demonstrated in several types of cancers and clinical trials are underway to see if inhibition of mTOR will be of benefit to a variety of cancer patients. A recent trial showed efficacy of RAD001 in advanced renal cancer (Hudes, et al. 2007).
All of these studies will be performed on an outpatient basis.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Yes/No (Response of "no" = patient ineligible)
Exclusion Criteria:
Yes/No (Response of "yes" = patient ineligible)
Patients who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as:
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.
Contact: Elliott Rudisill | 801-587-4765 | elliott.rudisill@hci.utah.edu |
Contact: Jessica Moehle | 801-587-4438 | jessica.moehle@hci.utah.edu |
United States, Utah | |
Huntsman Cancer Institute | Recruiting |
Salt Lake City, Utah, United States, 84112 |
Principal Investigator: | Randall W Burt, MD | Huntsman Cancer Institute |
Study Chair: | Scott Kuwada, MD | University of Hawaii |
Responsible Party: | Huntsman Cancer Institute ( Randall Burt, MD ) |
Study ID Numbers: | HCI # 26943 |
Study First Received: | November 17, 2008 |
Last Updated: | December 18, 2008 |
ClinicalTrials.gov Identifier: | NCT00811590 |
Health Authority: | United States: Food and Drug Administration |
Cancer Polyps |
Everolimus Sirolimus Hyperpigmentation Lentigo Skin Diseases Gastrointestinal Diseases Pigmentation Disorders Polyps |
Peutz-Jeghers Syndrome Intestinal Diseases Neoplastic Syndromes, Hereditary Digestive System Diseases Genetic Diseases, Inborn Peutz Jeghers syndrome Melanosis |
Neoplasms Pathologic Processes Disease Intestinal Polyposis Immunologic Factors |
Syndrome Physiological Effects of Drugs Immunosuppressive Agents Pharmacologic Actions |