Phase II Study of the Safety, Pharmacokinetics, and Exploratory Activity of Once Daily (QD) Topical Application of NS2 Cream to Treat Ichthyosis in Subjects With Sjögren-Larsson Syndrome (SLS)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- Number of Participants Experiencing a Serious Adverse Event (SAE).
研究概览
简要总结
This is a multi-center, randomized, double-blind, vehicle-controlled, parallel-group study designed to evaluate the safety, pharmacokinetic (PK), and exploratory activity of topically-applied NS2 dermatologic cream administered once-daily (QD) to subjects with ichthyosis secondary to Sjögren- Larsson Syndrome (SLS).
NS2 is expected to trap fatty aldehydes that are pathogenic in SLS patients, and thereby diminish the lipid-aldehyde adduct formation that likely results in ichthyosis associated with SLS, and potentially reduce the mild dermal inflammation characteristic of SLS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically-confirmed diagnosis of SLS
- •Active ichthyosis on the lower extremities that is determined to be at least moderate severity
排除标准
- •Evidence of an active infection
- •Currently receiving immunosuppressive therapy, including intermittent or low-dose corticosteroids and is not able or willing to suspend from 2 weeks before and during the study
- •Currently receiving systemic or topical retinoids, other topically applied drugs, or other supplements that could interfere with dermatologic examination findings
- •Received an investigational systemic or topically administered drug within 30 days before screening
研究组 & 干预措施
Active topical NS2 1% dermatologic cream
NS2 1% topical cream for dermal application
干预措施: Active topical NS2 1% dermatologic cream (Drug)
Topical vehicle dermatologic
Vehicle placebo for dermal application
干预措施: Vehicle placebo 0.0% NS2 dermatologic cream (Drug)
结局指标
主要结局
Number of Participants Experiencing a Serious Adverse Event (SAE).
时间窗: The safety assessment period is approximately 9 weeks.
Number of Participants Experiencing an Adverse Event Leading to Discontinuation.
时间窗: The safety assessment period is approximately 9 weeks.
次要结局
未报告次要终点