A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors (KCOT)
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|ClinicalTrials.gov Identifier: NCT02366312|
Recruitment Status : Active, not recruiting
First Posted : February 19, 2015
Last Update Posted : December 6, 2017
|Condition or disease||Intervention/treatment||Phase|
|Keratocystic Odontogenic Tumor||Drug: vismodegib||Phase 2|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||2 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Estimated Primary Completion Date :||May 2019|
|Estimated Study Completion Date :||May 2019|
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 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
- 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.
- 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.
- 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.
- 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.
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 ClinicalTrials.gov identifier (NCT number): NCT02366312
|United States, New York|
|NYU Bluestone Center For Cllinical Research|
|New York, New York, United States, 10010|
|Principal Investigator:||Brian L Schmidt, DDS, MD, PhD||NYU College of Dentistry|