This trial is active, not recruiting.

Condition keratocystic odontogenic tumor
Treatment vismodegib
Phase phase 2
Target PTCH1
Sponsor The Bluestone Center for Clinical Research
Collaborator Genentech, Inc.
Start date June 2016
End date May 2019
Trial size 2 participants
Trial identifier NCT02366312, 15-00254, ML28859


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
The 150-mg vismodegib drug product is a hard gelatin capsule formulation for oral administration. This study involves one year of treatment with Erivedge (150 mg/day) plus two years of follow-up.
vismodegib ERIVEDGE®, GDC-0449
vismodegib is a synthetic, small molecule inhibitor of the sonic Hh pathway, which is involved in tumorigenesis, thus providing a strong rationale for its use in the treatment of a variety of cancers.

Primary Outcomes

KCOT volume
time frame: 3 years
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
time frame: 3 years

Secondary Outcomes

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

Eligibility Criteria

Male or female participants at least 18 years old.

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).

Additional Information

Official title A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Principal investigator Brian L Schmidt, DDS, MD, PhD
Description 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).
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by The Bluestone Center for Clinical Research.