Keratocystic Odontogenic TumorPhase 2KCOTNCT02366312
A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
A Phase 2 study testing vismodegib in people with Keratocystic Odontogenic Tumor, run by NYU College of Dentistry at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Keratocystic Odontogenic Tumor
- What is being tested?
- vismodegib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Oct 2015to Aug 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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;
Who cannot join
- 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;
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- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
NYU Bluestone Center For Cllinical Research
New York, New York, 10010
About this study
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.
Read the 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).
- Conditions
- Keratocystic Odontogenic Tumor
- Being tested
- vismodegib
- Keywords
- Keratocystic odontogenic tumor, KCOT
- Medical subject headings
- Odontogenic Cysts
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
vismodegibDrug
Also called 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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: vismodegib |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Feb 2015 (estimated)
- Study start27 Oct 2015
- Primary completionAug 2019
- Study completionAug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Keratocystic Odontogenic TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU College of Dentistry
- Sponsor type
- University, hospital or other
- Collaborators
- Genentech, Inc.
- Responsible party
- Principal investigator, Brian L. Schmidt, NYU College of Dentistry
- Investigators
- Brian L Schmidt, DDS, MD, PhD, Principal investigator, NYU College of Dentistry
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02366312
- Sponsor's protocol ID
- 15-00254
- Other IDs
- ML28859
- Acronym
- KCOT
- Status last verified
- Feb 2021
- First submitted
- 5 Feb 2015
- Last updated
- 18 Feb 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
- ClinicalTrials.gov ID
- NCT02366312
- Acronym / sponsor ID
- KCOT / 15-00254
- Conditions
- Keratocystic Odontogenic Tumor
- Keywords
- Keratocystic odontogenic tumor, KCOT
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- NYU College of DentistryUniversity, hospital or other
- Collaborators
- Genentech, Inc.
- Study start
- 27 Oct 2015 (actual)
- Primary completion
- Aug 2019 (actual)
- Study completion
- Aug 2019 (actual)
- First posted
- 19 Feb 2015 (estimated)
- Results
- Posted 18 Feb 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| vismodegib | Experimental | Drug: vismodegibThe 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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;
Key exclusion criteria (12 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| vismodegibAlso: ERIVEDGE®, GDC-0449vismodegib 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. | Drug | vismodegib |
Primary outcome
KCOT Volume (Greatest Dimension)
Time frame: Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Feb 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Brian L Schmidt, DDS, MD, PhD principal investigator
NYU College of Dentistry
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing vismodegib in people with Keratocystic Odontogenic Tumor, run by NYU College of Dentistry at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 18 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
KCOT Volume (Greatest Dimension) (time frame: Baseline, 6 month (in treatment), 1 year (in treatment), 2 Year (post-treatment), 3 Year (post-treatment)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Feb 2021.