Clinical Trial: A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Brief Summary: The purpose of this study is to determine how well a daily dose of 150 mg of 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.
Detailed Summary:
This is a single-center, interventional, single-arm, open-label, two-cohort clinical trial. A total of 20 patients will be enrolled (10 with NBCCS-associated KCOT and 10 with sporadic KCOT) over a period of 2 years. This is a 3 year study where each patient will undergo up to 1 year of treatment and up to 2 years of post-treatment follow-up.
All patients will be assessed for safety and efficacy of the study drug GDC-0449 (Vismodegib).
Sponsor: The Bluestone Center for Clinical Research
Current Primary Outcome:
- KCOT volume [ Time Frame: 3 years ]
- Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 3 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Minimal clinically significant changes in the tumor vs. theoretical expectation of 5 % improvement [ Time Frame: 3 years ]
- PTCH1 mutation effect on tumor volume [ Time Frame: 3 years ]
Original Secondary Outcome: Same as current
Information By: The Bluestone Center for Clinical Research
Dates:
Date Received: February 5, 2015
Date Started: June 2016
Date Completion: May 2019
Last Updated: July 22, 2016
Last Verified: July 2016
