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

NCT02366312 | Completed Phase 2 Results DMC

• 2 other identifiers: 15-00254ML28859
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

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.

Conditions

Keratocystic Odontogenic Tumor

Keywords

Keratocystic odontogenic tumor; KCOT

Outcome Measures

Primary Outcomes (1)

• KCOT Volume (Greatest Dimension)

  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.

  Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)

Study Arms (1)

vismodegib

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.

Drug: vismodegib

Interventions

vismodegib DRUG

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.

Also known as: ERIVEDGE®, GDC-0449

vismodegib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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;

You may not qualify if:

• 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).

Sponsors & Collaborators

• NYU College of Dentistry: lead
• Genentech, Inc.: collaborator

Study Sites (1)

NYU Bluestone Center For Cllinical Research

New York, New York, 10010, United States

Location

MeSH Terms

Conditions

Odontogenic Cysts

Interventions

HhAntag691

Condition Hierarchy (Ancestors)

Jaw Cysts; Bone Cysts; Cysts; Neoplasms; Jaw Diseases; Musculoskeletal Diseases; Stomatognathic Diseases

Limitations and Caveats

Enrollment for the study closed early by the sponsor due to low enrollment number. Only two subjects were enrolled and received the study treatment.

Results Point of Contact

• Title: Dr. Brian Schmidt
• Organization: NYU College of Dentistry

brianl.schmidt@nyu.edu

212-998-8900

Study Officials

• Brian L Schmidt, DDS, MD, PhD

  NYU College of Dentistry

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Bluestone Center for Clinical Research

Study Record Dates

• First Submitted: February 5, 2015
• First Posted: February 19, 2015
• Study Start: October 27, 2015
• Primary Completion: August 1, 2019
• Study Completion: August 1, 2019
• Last Updated: February 18, 2021
• Results First Posted: February 18, 2021

Record last verified: 2021-02

Locations

US (1)