Clinical trial NCT02366312: A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Official title: A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Keratocystic Odontogenic Tumor
Keywords: Keratocystic odontogenic tumor
KCOT
Lead sponsor: NYU College of Dentistry
Sponsor class: OTHER
Collaborators: name: Genentech, Inc.
First posted: 2015-02-19
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 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).

Eligibility 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).

Primary outcomes:
measure: KCOT Volume (Greatest Dimension)
timeFrame: Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: NYU Bluestone Center For Cllinical Research
state: New York
Study officials:
affiliation: NYU College of Dentistry
name: Brian L Schmidt, DDS, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: