A Multicenter, Randomized, Double Blind, Vehicle-controlled, Phase 3 Efficacy and Safety Study of Patidegib Gel 2% for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas (BCCs) in Subjects With Gorlin Syndrome
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 113
- 试验地点
- 58
- 主要终点
- Number of new BCCs on the face at Month 12
研究概览
简要总结
The aim of this clinical study is to find out how well Patidegib Gel 2% works in preventing new basal cell carcinomas (BCCs) developing on the face of adults with Gorlin syndrome, and how safe Patidegib Gel 2% is to use.
People who take part will apply either Patidegib Gel 2% or a Vehicle Gel (with no active drug substance) to their face twice a day for a year (12 months). The number of new BCCs on the face will be compared between those who used Patidegib Gel 2% or Vehicle Gel after 12 months.
详细描述
This is a global, multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Gel 2%, applied topically twice daily to the face of adult subjects with Gorlin syndrome.
Subjects will be randomized (1:1) to receive either Patidegib Gel 2% or Vehicle Gel for 12 months.
The assignment of subjects to the 2 groups will be stratified by sex assigned at birth, age (≥60 or <60 years), and number of BCC lesions at the treatment area (face) (10-15, 16-30 or >30) at Baseline.
All suspicious lesions will be imaged and tracked consistently throughout the study so that new BCCs that arise can be readily identified. New BCCs will be confirmed by dermoscopic analysis through the study. The BCC images will be reviewed and assessed by a Central Photo Review Board (CPRB).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •The subject must be at least 18 years old at the Screening Visit.
- •The subject must be confirmed to have a PTCH1 mutation.
- •The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1).
- •The subject must be willing to abstain from application of any non-study topical medication (prescription or over the counter) to their facial skin for the duration of the trial, except as prescribed by the investigator.
排除标准
- •The subject has used topical treatment to the face or systemic therapies that might interfere with the evaluation of the study Investigational Product (IP).
- •The subject is known to have hypersensitivity to any of the ingredients in the IP formulation.
- •The subject has uncontrolled systemic disease.
- •The subject has been treated for invasive cancer within the past 5 years excluding chronic lymphocytic leukemia Stage 0, non-melanoma skin cancer, successfully treated melanoma in situ and Stage I melanoma, Stage I cervical cancer, or ductal carcinoma in situ of the breast.
- •Inefficacy of previous Hedgehog inhibitor therapy.
研究组 & 干预措施
Patidegib Gel 2%
Patidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months
干预措施: Patidegib Topical Gel (Drug)
Vehicle Gel
Vehicle Gel, applied topically to the face twice daily for 12 months
干预措施: Patidegib Topical Gel with no active patidegib (Drug)
结局指标
主要结局
Number of new BCCs on the face at Month 12
时间窗: Month 12
New BCCs at Month 12 in the Patidegib Gel 2% and Vehicle Gel groups, as assessed by the CPRB
次要结局
未报告次要终点