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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02366312
Recruitment Status : Completed
First Posted : February 19, 2015
Last Update Posted : August 26, 2019
Genentech, Inc.
Information provided by (Responsible Party):
Brian L. Schmidt, NYU College of Dentistry

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.

Condition or disease Intervention/treatment Phase
Keratocystic Odontogenic Tumor Drug: vismodegib Phase 2

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).

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 2 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
Actual Study Start Date : October 27, 2015
Actual Primary Completion Date : August 2019
Actual Study Completion Date : August 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Vismodegib

Arm Intervention/treatment
Experimental: vismodegib
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.
Drug: vismodegib
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.
Other Name: ERIVEDGE®, GDC-0449

Primary Outcome Measures :
  1. KCOT volume [ Time Frame: 3 years ]
    To evaluate the efficacy of GDC-0449 in reducing KCOT size (shrinkage) in NBCCS-associated KCOT and sporadic KCOT patients following 6 to 12 months of ingestion of 150 mg/day, up to 1 year of treatment and up to 2 years of post- treatment follow-up.

  2. Number of Participants with Adverse Events as a Measure of Safety and Tolerability [ Time Frame: 3 years ]
    To evaluate the safety of this dose of GDC-0449 in these patients.

Secondary Outcome Measures :
  1. Time of response of a GDC-0449 anti-proliferative effect [ Time Frame: 3 years ]
    To determine the time of response of a GDC-0449 anti-proliferative effect (i.e. tumor shrinkage) after administration of the drug up to one year or until treatment is stopped.

  2. PTCH1 mutation effect on tumor volume [ Time Frame: 3 years ]
    To assay germ line PTCH1 mutations before treatment with GDC-0449 in patients with NBCCS-associated KCOT.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02366312

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United States, New York
NYU Bluestone Center For Cllinical Research
New York, New York, United States, 10010
Sponsors and Collaborators
NYU College of Dentistry
Genentech, Inc.
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Principal Investigator: Brian L Schmidt, DDS, MD, PhD NYU College of Dentistry

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Responsible Party: Brian L. Schmidt, Director, Bluestone Center for Clinical Research, NYU College of Dentistry Identifier: NCT02366312    
Other Study ID Numbers: 15-00254
ML28859 ( Other Identifier: Genentech )
First Posted: February 19, 2015    Key Record Dates
Last Update Posted: August 26, 2019
Last Verified: August 2019
Keywords provided by Brian L. Schmidt, NYU College of Dentistry:
Keratocystic odontogenic tumor
Additional relevant MeSH terms:
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Odontogenic Tumors
Neoplasms by Histologic Type