A Pilot Study to Investigate the Off Label Use of Vismodegib as an Adjuvant to Surgery for Basal Cell Carcinoma Tumors (BCCs)
试验速览
- 阶段
- 早期 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated
研究概览
简要总结
The purpose of this study is to learn about the effect of vismodegib on sporadic basal cell carcinoma (BCCs) prior to surgical removal.
详细描述
PRIMARY OBJECTIVES:
I. The percent reduction in surgical defect area/size surrounding BCC tumor pre and post-vismodegib.
SECONDARY OBJECTIVES:
I. Recurrence rate post treatment II. Safety, tolerability and percent drop-out after 3 vs. 6 months of vismodegib in otherwise healthy patients.
OUTLINE:
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
- •Study patients must have at least one BCC, > 5 mm, eligible for Mohs surgical removal; patients with BCCs that have been treated before (recurrent BCCs, BCCs that failed other chemotherapy) are eligible for this trial, if they meet size criteria
- •No Eastern Cooperative Oncology Group (ECOG) or Karnofsky performance status will be employed
- •Normal hepatic function: aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2 x the upper limit of normal (ULN)
- •Normal renal function : normal serum creatinine defined as <= 2.5 mg/dL
- •Clinically acceptable complete blood count (CBC)
- •Ability to understand and the willingness to sign a written informed consent document
- •The patient is willing to forego surgical treatment of BCCs by up to 6 months, except when the principal investigator (PI) believes that delay in treatment potentially might compromise the health of the subject
- •Documented negative serum pregnancy test for women of childbearing potential, with agreement to the use of two acceptable methods of contraception during the study and for 7 months after discontinuation of vismodegib
- •For men with female partners of childbearing potential, agreement to use a latex, non-latex, or any other male condom and to advise their female partners to use an additional acceptable method of birth control during the study and for 2 months after discontinuation of study drug
- •Be willing to not donate blood or semen for three months following discontinuation of study medications
排除标准
- •The patient has a history of invasive cancer within the past five years excluding non-melanoma skin cancer, stage I cervical cancer, ductal carcinoma in situ of the breast, or chronic lymphocytic leukemia (CLL) stage 0
- •The subject has uncontrolled systemic disease, including known human immunodeficiency virus (HIV) positive patients:
- •The patient has history of congestive heart failure
- •The patient has clinically important history of liver disease, including viral or hepatitis, current alcohol abuse, or cirrhosis
- •The patient has any condition or situation which in the investigator's opinion may put the patient at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study; this includes history of other skin conditions or disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding giving reasonable suspicion of a disease or condition that contraindicates use of an investigational drug or that might affect interpretation of the results of the study or render the patient at high risk from treatment complications
- •The patient has a history of hypersensitivity to any of the ingredients in the study medication formulations
- •The patient is willing to abstain from application of non-study topical medications to the skin for the duration of the study, including prescription and over the counter preparations; for example, topical preparations containing corticosteroids or vitamin A derivatives are not allowed
- •Pregnant or nursing patients will be excluded from the study
研究组 & 干预措施
Treatment (vismodegib and Mohs surgery)
Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.
干预措施: vismodegib (Drug)
Treatment (vismodegib and Mohs surgery)
Patients receive vismodegib PO daily for 3-6 months based on the size of basal cell carcinoma and then undergo Mohs surgery.
干预措施: Mohs surgery (Procedure)
结局指标
主要结局
Percent Change in Surgical Defect Area After the Treatment Period Using Calipers and Photographs Was Calculated
时间窗: average of 4 months
At baseline, we selected 1 to 2 tumors per patient for surgery (13 target tumors selected). At baseline,1 Mohs surgeon measured the estimated surgical defect area around the target tumor. For tumors to be excised by Mohs we defined estimated surgical defect as the tumor size plus a 2-mm circumferential margin, presuming tumor clearance after a Mohs stage-1 excision. For the tumor undergoing standard (non-Mohs) excision, we used tumor size plus a standard 4-mm margin11 for the estimated surgical defect. On the day of the surgery, we measured the surgical defect area as the final tumor-free defect after the Mohs procedure or non-Mohs excision immediately before closure. We used the Image J software program (National Institutes of Health, Bethesda, MD) to calculate tumor area (cm2). Only target tumors are included in this analysis.
次要结局
- Number of Tumors Demonstrating Histologic Cure(Average of 4 months)
- Tumor Recurrence Rate of Treated BCCs(average of 22 months)
- Tumor Size Measurements Before and After Short Term Vismodegib Treatment(4 months (average))
研究者
Jean Yuh Tang
Associate Professor of Dermatology
Stanford University