A Randomized, Parallel, Double-Blind, Vehicle Controlled Study to Evaluate the Safety and Efficacy of Two Concentrations of Topical TMB-001 for the Treatment of Congenital Ichthyosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- LEO Pharma
- 入组人数
- 34
- 试验地点
- 22
- 主要终点
- Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement
研究概览
简要总结
The purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1% isotretinoin) in subjects 9 years of age and older for the treatment of congenital ichthyosis (CI), including recessive X-linked ichthyosis (RXLI) and autosomal recessive congenital ichthyosis-lamellar ichthyosis (ARCI-LI) subtypes.
Funding Source FDA-OOPD
详细描述
This is a randomized, parallel, double-blind, vehicle-controlled study to evaluate the safety and efficacy of two concentrations of topical TMB-001 for the treatment of CI in subjects with either the ARCI-LI or RXLI subtypes. The duration of treatment will be 12 weeks. Each subject will participate in the study for up to 24 weeks (including up to a 90-day Screening period). Eleven study centers from United States of America and Australia participated in this global study. Each subject participated in the study for up to 24 weeks (including up to a 90-day Screening period).
Eligible subjects were randomized (1:1:1) to one of 3 treatment groups:
- TMB-001, 0.05%, twice daily (bid)
- TMB-001, 0.1%, bid
- Vehicle Ointment (Control), 0%, bid
Subjects were evaluated on a monthly basis for safety and efficacy results over the 12- week treatment period. Efficacy was determined in 2 ways:
- VIIS (or Visual Index of Ichthyosis Severity) "treatment success" (VIIS-50) was defined as ≥ 50% reduction from Baseline for VIIS scaling score for the sum of the scores for 'VIIS body areas' that had a Baseline score ≥ 3). The proportion of subjects achieving VIIS-50 at Visit 6 at Week 12 relative to Baseline was the primary efficacy endpoint and time point used to compare the two active treatments to vehicle.
- The proportion of subjects achieving IGA (Investigator Global Assessment) treatment success will be the key secondary endpoint. For this endpoint, the IGA score was established as "treatment success" or "treatment failure" where "treatment success" is defined as at least a 2-grade decrease in severity score (for example, severe or score of 4 at baseline to mild or score of 2 at Visit 6) relative to Baseline at Visit 6 at week 12.
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)
盲法说明
Double-blind
入排标准
- 年龄范围
- 9 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject is male or female, 9 years of age or older at Visit 2 (Baseline)
- •Subject has provided written informed consent
- •Females of child bearing potential must be surgically sterile or agree to 2 forms of birth control
- •Subject has clinical diagnosis of Congenital Ichthyosis with genetic confirmation of subtype
- •Subject has between 10% and 90% total BSA affected by Congenital Ichthyosis
- •Subject has at least 2 VIIS assessment areas with a scaling score of 3 or greater
排除标准
- •Subject has inflammatory skin diseases unrelated to ichthyosis
- •Subject has used other prohibited topical treatments in the assessment areas within certain days from baseline
- •Subject has used systemic retinoids within12 weeks of baseline
- •Subject has untreated secondary infections
- •Subject has lesions suspicious for skin cancer or untreated skin cancers
研究组 & 干预措施
TMB-001 Ointment, 0.1%
Topical, BID (Twice daily)
干预措施: Isotretinoin (Drug)
TMB-001 Ointment, 0.05%
Topical, BID ( twice daily)
干预措施: Isotretinoin (Drug)
Vehicle Ointment (Control)
Topical, BID (Twice daily)
干预措施: Vehicle (Other)
结局指标
主要结局
Number of Subjects With Reduction in Targeted Ichthyosis Severity Using the Visual Index for Ichthyosis Severity Measurement
时间窗: 12 weeks
Number of subjects with Visual Index for Ichthyosis Severity (VIIS) treatment success, defined as 50% or greater decreased in VIIS scaling score
次要结局
- Number of Subjects With Reduction in Overall Ichthyosis Severity as Measured With Investigator Global Assessment(12 weeks)
- Change in I-NRS (Itch-Numeric Rating Scale) From Baseline at Week 12(12 weeks)
- Change From Baseline in Dermatology Life Quality Index (DLQI) at Week 12(12 weeks)