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Pilot Study of mTOR Inhibitor Therapy in Peutz-Jeghers Syndrome

NCT00811590 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2024-09-19

Study results available
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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.

Conditions

  • Peutz-Jeghers Syndrome

Interventions

DRUG

Everolimus

Everolimus is a novel derivative of rapamycin. Everolimus 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. Everolimus is being investigated as an anticancer agent based on its potential to act * directly on the tumor cells by inhibiting tumor cell growth and proliferation * indirectly by inhibiting angiogenesis leading to reduced tumor vascularity (via potent inhibition of tumor cell VEGF production and VEGF-induced proliferation of endothelial cells.

Sponsors & Collaborators

Principal Investigators

  • Randall W Burt, MD · Huntsman Cancer Institute

  • Scott Kuwada, MD · University of Hawaii

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2008-11-30
Primary Completion
2011-03-31
Completion
2011-03-31

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00811590 on ClinicalTrials.gov