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

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;