Overview

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

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.

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
Arm
(Experimental)
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

Measure
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

Measure
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.