A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors

• Status: Completed
• Trial end date: 2019-08-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: NYU College of Dentistry; The Bluestone Center for Clinical Research
• Collaborator: Genentech, Inc.

Criteria

Inclusion Criteria:

• Males and females, 18 years of age and above at the time the informed consent form is signed;

• Able to understand and sign the Informed Consent Form and other necessary paperwork prior to initiation of study procedures;

• Able to communicate with the investigator/study site personnel, understand and comply with the study requirements, and willing to return for specified visits at the appointed time;

• Patients who have received prior treatment for their KCOT and with a diagnosis of recurrent (maxillary or mandibular) sporadic KCOT or NBCCS-associated KCOT (single or multiple);

• Diagnosis of KCOT will be done by past pathology report or by biopsy at the study site, if applicable;

• Willingness to consent to biopsy of the lesion, if needed;

• Willingness to delay excision of the target tumor site, unless evidence of disease progression or lack of drug tolerability;

• Willingness to donate blood for genetic testing;

• For female patients of childbearing potential, agreement to use two acceptable methods of birth control, including one barrier method during the study and 7 months after discontinuation of study drug;

• For males with female partners of childbearing potential, agreement to use a male condom (with spermicide) and to advise their female partners to use an acceptable method of birth control during the study and for 2 months after the discontinuation of the study drug;

• Agreement not to donate blood/blood products during the study and for 7 months after the discontinuation of the study drug;

• For males not to donate sperm products or semen during treatment and for 2 months after the discontinuation of the study drug;

• Able and willing to swallow pill;

• No malabsorption syndrome or other condition that would interfere with enteral absorption;

• At least 4 weeks since last chemotherapy, investigational therapy, radiotherapy or major surgical procedure and recovered from the first study drug administration;

• KCOT measures at least 1 cm in one dimension on pretreatment volumetric CT scan;

• No clinically significant abnormalities with clinical laboratory assessments;

Exclusion Criteria:

• Concurrent anti-tumor therapy;

• Completion of the most recent anti-tumor therapy (including Vismodegib) less than 4 weeks prior to the initiation of treatment (first study drug administration);

• Uncontrolled medical illness;

• Pregnancy or lactation; female patients who are planning to become pregnant for the duration of the study and 7 months post-treatment;

• Inability or unwillingness to swallow capsules;

• Any medical or psychological illness or condition preventing adequate consent;

• History of significant atherosclerotic disease, including the following:

• Coronary artery disease (i.e., myocardial infarction within the past year or unstable angina);

• Documented carotid atheroma;

• Known HIV infection;

• Current alcohol abuse;

• History of resistance to vismodegib (patients who previous received vismodegib for BCC and had no clinical response will be excluded).