A Randomized,Single-Blind, Placebo-controlled, Self-matched Pairing, Independent Evaluated Study to Evaluate the Efficacy and Safety of RGN-137 Topical Gel in Subjects With Junctional and Dystrophic Epidermolysis Bullosa (CELEB)
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 4
- 试验地点
- 5
- 主要终点
- Time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84.
研究概览
简要总结
The objective of this study is to compare the efficacy and safety of RGN-137 topical gel with that of placebo gel for treatment of junctional epidermolysis bullosa (JEB) or dystrophic epidermolysis bullosa (DEB).
详细描述
RGN-137 will be evaluated for efficacy and safety compared to a Placebo. A matched-pair design will be used to evaluate RGN-137 treatment versus placebo for treatment of 15 subjects with JEB or DEB. Eligible subject must have 1 set of matched-pair wound. The investigator will assign pair of index wounds, each wound with an area between 5cm2 and 50cm2, inclusive, for the eligible subject on Day 1, and for each pair, one wound will be randomized to receive RGN-137 gel and the other to receive Placebo gel. Subjects and independent evaluator will be blinded to the treatment assignments for each wound.
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
- Triple (Participant, Care Provider, Outcomes Assessor)
入排标准
- 年龄范围
- 4 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female and at least 4 years old with a diagnosis of DEB or JEB
- •Patients and their parents or guardians must be willing and able to provide written informed consent/assent
- •Presence of 1 pair of stable index wounds within the specified size range at study
- •Index wounds must have a surface area between 5 cm2 and 50 cm2, inclusive at the screening visit
- •The index wounds selected as pairing must be relatively matched in terms of size and location
- •Women of childbearing potential must have a negative pregnancy test prior to randomization
- •Sexually active subjects must agree to use medically accepted methods of contraception during the study
排除标准
- •Have any clinical evidence of local infection of the index lesion
- •Use of prior or concomitant medication (Any investigational drug within 30 days, Immunotherapy or cytotoxic chemotherapy within 60 days, Systemic steroidal therapy within 30 days, and topical steroidal therapy within 14 days)
- •History of sensitivity to any component of the treatment
- •Neurological, cardiovascular, respiratory, hepatic, renal, or metabolic disease likely to interfere with the subject's participation in or completion of the study (At the discretion of investigator, participants with cardiomyopathy can participate)
- •Current or former malignancy, including a history of squamous cell carcinomas
- •Arterial or venous disorder resulting in ulcerated wounds
- •Uncontrolled diabetes mellitus
- •Pregnancy or breastfeeding during the study
- •Girls or women who have had menarche but have not completed menopause
- •Any condition or situation likely to cause the subject to be unable or unwilling to participate in the study procedures
研究组 & 干预措施
RGN-137
It is formulated as a gel for topical administration.
干预措施: RGN-137 (Drug)
Placebo
It is composed of the same excipients as RGN-137 formulation without the active ingredient.
干预措施: Placebo (Drug)
结局指标
主要结局
Time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84.
时间窗: up to Day 84
This primary endpoint will the time to achieving 50% reduction in area of each index wound from Day 1 up to Day 84, defined as date of the first study visit on which 50% reduction in index wound area is achieved minus date of Day 1 visit.
次要结局
- Time to achieving 50%, 75%, and 100% (with drainage or without drainage) reduction in area of each index wound from Day 1 up to Day 84.(up to Day 84)
- Incidence of 50%, 75%, and 100% reduction and complete re-epithelialization without drainage in area of each index wound at the scheduled visits.(up to Day 84)
- Change from Baseline and percent change from Baseline in surface area of each index wound at the scheduled visits.(up to Day 84)