A Phase 2 Randomized, Multicenter, Double-blind, Vehicle Controlled, 90-Day, Safety, Efficacy & Systemic Exposure Study of Trifarotene (CD5789) Cream HE1 in Adults and Adolescents With Autosomal Recessive Ichthyosis With Lamellar Scale
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 65
- 试验地点
- 36
- 主要终点
- The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.
研究概览
简要总结
This is a phase 2 randomized, multi-center, double-blind, vehicle controlled, 90 day, safety, efficacy, and systemic exposure study followed by a 90 day open-label extension of trifarotene cream in adults and adolescents with autosomal recessive ichthyosis with lamellar scale.
详细描述
This is a 2-cohort, multicenter study in subjects with moderate to severe LI. Adults (Cohort A) and adults and adolescents (Cohort B) will be randomized in a double-blind fashion to 1 of 2 doses of active or vehicle and treated twice weekly for 90 days. Subjects who complete the randomized, double-blind portion of the study will be eligible to enter a 90 day, open-label extension study.
Approximately 15 adults (≥18 years old) will be randomized into the first cohort of subjects (Cohort A) in a 1:1:1 ratio and treated twice weekly for up to 90 days. If no safety issues are identified, both adults and adolescents (ages 12-17 years, inclusive) will be allowed to enroll in Cohort B. Subjects in Cohort B will be randomized 1:1:1 and treated twice weekly for up to 90 days in the same manner as subjects in Cohort A.
All subjects who complete 90 days of double-blind study treatment will be eligible to enroll in a 90 open-label extension. Subjects in the open-label extension will receive active twice weekly for up to 90 days.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
CD5789 Cream 200 µg/g
CD5789 200 µg/g, topical, 50g
干预措施: CD5789 Cream 200 µg/g (Drug)
CD5789 Cream 100 µg/g
CD5789 100 µg/g, topical, 50g
干预措施: CD5789 Cream 100 µg/g (Drug)
CD5789 Cream Vehicle
CD5789 Cream Vehicle, topical, 50g
干预措施: CD5789 Cream Vehicle (Drug)
结局指标
主要结局
The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.
时间窗: 90 Days
The percentage of subjects in each treatment group who experienced successful resolution of LI where "success" is defined as clear/almost clear on treated areas and at least a 2-grade change from Baseline at Day 90/end-of-treatment (EOT) in the Double-blind Period on the 5-point IGA full body scale.
次要结局
- The Difference in Mean Scores Using Individual Score for Roughness(90 Days)
- The Difference in Mean Scores Using Palm Sole Assessment(90 Days)
- Total 16-point Visual Index for Ichthyosis Severity (VIIS)(90 Days)
- The Difference in Proportion of Subjects With Presence of Fissures on Soles Between the Active and Vehicle Groups(90 Days)
- Quality of Life Measurement Per Dermatology Life Quality Index (DLQI)(90 Days)
- The Difference in Proportion of Subjects With Presence of Fissures on Palms Between the Active and Vehicle Groups(90 Days)