A Phase 2, Single-center, Single-arm, Open Label Trial of Vismodegib in Patients With Keratocystic Odontogenic Tumors
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- KCOT Volume (Greatest Dimension)
研究概览
简要总结
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.
详细描述
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).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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;
排除标准
- •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).
研究组 & 干预措施
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.
干预措施: vismodegib (Drug)
结局指标
主要结局
KCOT Volume (Greatest Dimension)
时间窗: 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.
次要结局
未报告次要终点
研究者
Brian L. Schmidt
Director, Bluestone Center for Clinical Research
NYU College of Dentistry