A Multicenter, Phase 2B, Randomized, Double-Blind, Stratified, Vehicle-Controlled Study Evaluating The Safety And Efficacy Of QTORIN 3.9% Sirolimus Topical Gel In Prevention Of BCCs In Patients With Gorlin Syndrome
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 73
- 试验地点
- 16
- 主要终点
- Incidence of dermatological, treatment emergent adverse events after treatment with active
研究概览
简要总结
A Phase 2b study looks at the safety and efficacy of a treatment that is being investigated for people with certain diseases. This study will be conducted at multiple centers in the United States where participants with Gorlin Syndrome, also known as basal cell nevus syndrome, will be randomly placed into two groups; one group will receive the active topical gel, the other a topical vehicle gel, also know as placebo. Participants will apply this topical product to their face once a day for 6 months. The study will be looking at the number of new BCCs that develop on the faces of all the participants during this time.
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
- •Adults, 18 years or older
- •Meet diagnostic criteria for Gorlin Syndrome
- •Willing to avoid application of a non-study topical medication to the face (prescription or over the counter) during the study.
- •Willing to forego treatment of BCCs with anything other than the study medication except when the Investigator believes that delay of treatment of a BCC potentially might compromise the health of the subject. During the trial, the only allowed form of BCC treatment is surgical.
- •Participant will be encouraged to use their preferred sunscreen with a sun protector factor (SPF) of at least 30 daily
排除标准
- •Recently participated in a clinical trial evaluating an investigational product for the treatment of BCCs or Gorlin Syndrome within 3 months prior to to starting study medication. Participant can start the study screening period prior to completing the 3 month washout.
- •Recently used topical or systemic (oral) treatment that might interfere with the evaluation of the study medication.
研究组 & 干预措施
Active
干预措施: PTX-022 (Drug)
Vehicle
干预措施: Vehicle comparator (Drug)
结局指标
主要结局
Incidence of dermatological, treatment emergent adverse events after treatment with active
时间窗: Month 6
Number of new biopsy confirmed BCCs that develop on the face compared between the active and vehicle treatment arms
时间窗: Month 6
次要结局
- Percentage of participants developing 2 or more new biopsy confirmed BCCs on the face.(Month 6)
- Percentage of participants developing 1 or more new biopsy confirmed BCCs on the face.(Month 6)