2023-507528-21-00招募中3 期
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
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 60
- 试验地点
- 14
- 主要终点
- Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome as assessed by the CPRB
研究概览
简要总结
To assess the efficacy of Patidegib Gel 2% when applied twice daily to the face of subjects with Gorlin syndrome
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Remote safety follow-up Visit
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
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 and non-melanoma skin cancer, Stage I cervical cancer, or ductal carcinoma in situ of the breast
- •Previous Hedgehog inhibitor therapy was considered to have failed for reasons other than unfavorable side effect(s)
结局指标
主要结局
Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome as assessed by the CPRB
Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome as assessed by the CPRB
次要结局
- Number of New Surgically Eligible BCCs (nSEBs) at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome
- Change from Baseline to Month 12 in Advanced Basal Cell Carcinoma Index (aBCCdex) lesion symptoms scale score
- Change from Baseline to Month 12 in confirmed BCCs lesion diameter
- Proportion of Surgically Eligible BCCs (SEBs) and nSEBs assessed as resolved by Month 12
- Proportion of SEBs and nSEBs per subject that have regressed by Month 12
- Proportion of subjects developing ≥2 facial new BCCs at Month 12
- Change in total number of confirmed BCCs from Baseline to Month 12
- Change from Baseline to Month 12 in the Skin Cancer Index quality of life questionnaire score
- Adverse events (AEs), including serious adverse events (SAEs) occurring at any time during the study
- Investigator Dermal Safety and Local Tolerability Assessments score at any time during the study
- Change from Baseline to Month 12 in Patient Global Impression in disease Severity (PGI-S)
- Change in Patient Global Impression of Change in clinical status (PGI C) at Month 12
- Number of new BCCs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome
- Number of nSEBs at Month 12 in the 2 arms (Patidegib Gel 2% and vehicle) when applied twice daily to the face of subjects with Gorlin syndrome
- Time to first new BCC
- Skin pain and peak pruritus scores as reported by the subject on a numeric rating scale
研究者
Clinical team member
Scientific
Sol-Gel Technologies Ltd.
研究点 (14)
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