Bluestone Trials

Keratocystic Odontogenic Tumor (KCOT) Study

Seeking patients that have received prior treatment for KCOT and have a diagnosis of recurrent KCOT.
Status: Ongoing, enrollment of subjects completed.

Study Name

A phase 2, single-center, single-arm, open-label trial of vismodegib in patients with keratocystic odontogenic tumors

 

Short Study Description 

The purpose of this study is to determine how well a daily dose of 150 mg of the investigational drug, Erivedge (vismodegib), reduces keratocystic odontogenic tumor (KCOT) size, and to evaluate the safety of this dose.  Erivedge is FDA-approved for use in adults with a specific type of skin cancer. However, the drug is experimental for patients with KCOT. It is our hope that this research could help future patients who have recurring KCOT.  It is also possible that no benefit will come from this study.

If you are 18 and older and have received prior treatment for your KCOT and have a diagnosis of recurrent KCOT that measures at least 1 cm in one dimension, you may qualify to participate in a research study at the NYU Bluestone Center for Clinical Research.  Research participants must be willing to stay enrolled for the duration of the study.

 

Study Details

KCOT is a benign odontogenic neoplasm that has destructive and infiltrative behavior. Although KCOTs are benign, they tend to be aggressive, with local invasion of bony structures, extensive growth, and potential for substantial disfigurement. This condition has a high recurrence rate and it is estimated that after initial treatment with various procedures recurrence ranges from 2.5% to 62.5%. The only therapeutic option for patients affected by KCOT is surgery which can involve significant destruction of tissue; often multiple surgeries are required. Thus, the management of patients with KCOT has challenged and frustrated both patients and providers.

This study involves one year of treatment with Erivedge (150 mg/day) plus two years of follow-up. The first study visit may take place all at once or over the course of a few days. At this visit, we will make sure you are eligible to participate in the study and ask you to complete some baseline procedures.

During the second phase of the study (treatment), we will ask you to attend a study visit once a month for a year (12 visits total) while you are taking 150 mg/day of Erivedge. If you are not able to tolerate the drug due to the side effects or if the study investigator thinks it is in your best interest to temporarily stop taking the drug, you will be given a 1-3 month treatment break. If, after 3 months, you are not able to resume taking the study drug, you will be discontinued from the study.

During the third phase of the study (post-treatment), we will ask to you attend study visits at 1, 2, 3, and 7 months, and 1 and 2 years post-treatment.