An Open-label, Phase II Study to Evaluate the Clinical Efficacy and Safety of Tapinarof for Adult Patients With Palmoplantar Keratoderma
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Physician's Global Assessment (PGA)
研究概览
简要总结
The purpose of this study is to assess adults with palmoplantar keratoderma (thickening skin layer on palms and soles) who are treated with the study drug, tapinarof. This is a naturally occuring compound used for the treatment of psoriasis. This study is being done to find out how well and safe this drug is for stopping or treating keratoderma. This study aims to investigate the positive impacts of daily topical Tapinarof use in the improvement of Keratoderma. Clinical efficacy and safety profile of prescribing Tapinarof for this condition will be evaluated.
详细描述
This study aims to assess the benefits of the treatment. Should the treatment demonstrate benefits (as defined by the primary outcome measure) without any observed serious adverse events (SAEs), further clinical trials would be justified. If at least 2 out of 6 subjects benefits from the treatment (as defined by our primary outcome measure) and there are no SAEs, future clinical trials would be warranted. This initial data will determine whether it is worthwhile to proceed with a phase 2 trial, that will be both blinded and well-controlled.
The study is designed to gather preliminary experiences that can be used to design subsequent, well-controlled studies that are appropriately powered for statistical analysis. Currently, there is not enough experience with this medication in treating this specific condition, which is necessary to design successful randomized controlled trials (RCTs) for regulatory approval by the FDA. In dermatology, conducting studies in this manner is standard practice.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Ability to discern and provide written informed consent and voluntarily adhere to all of the protocol requirements
- •Male or female subjects in their adult years (≥ 18 years of age), inclusive, from the time of providing written subject-investigator agreement.
- •Confirmed clinical diagnosis of palmoplantar keratoderma.
排除标准
- •Treatment with any of the following medications and therapies during the duration of the study*:
- •Topical corticosteroids
- •Keratolytic agents (eg, 6% salicylic acid, 70% propylene glycol, 30% water)
- •Topical retinoids
- •Topical vitamin D ointment (calcipotriol)
- •Oral retinoids (acitretin)
- •If on any of these then a washout period of at least 2 weeks will be enforced before the study begins. This washout period is meant to ensure that any effects or residues from previous treatments are minimized before starting the study.
- •Prior exposure to Tapinarof treatment
- •Known history or suspicion of hypersensitivity reactions, or contraindications to any or all components of the drug or drugs of similar properties.
- •Currently participating in another clinical study for the same purpose.
- •Potential or confirmed pregnancy: Individuals who are currently pregnant or have confirmed pregnancy at the time of screening will be excluded from participation in the study. This criterion aims to ensure the safety of participants and adhere to ethical considerations regarding the potential risks associated with the study interventions during pregnancy.
- •Any other factors specifically not listed above but deemed patient unsuitable by the discretion of the investigators.
研究组 & 干预措施
Tapinarof Drug Treatment
Subjects in this arm will apply Tapinarof 1% cream topically once daily for a 16-week open-label treatment period. Participants who achieve disease clearance after 16 weeks may discontinue treatment and will be monitored for sustained response up to 52 weeks. Those who do not achieve clearance will continue the treatment during an extension period for up to 36 additional weeks or until clearance is achieved, whichever comes first. Regular follow-up visits will assess the duration of disease clearance and monitor for any potential recurrence of keratoderma.
干预措施: Tapinarof (Drug)
结局指标
主要结局
Physician's Global Assessment (PGA)
时间窗: Week 16 (Visit 4)
To evaluate the treatment benefit of Tapinarof applied topically to patients affected with palmoplantar keratoderma. Either an overall change in the PGA score to 0 (clear) or 1 (almost clear) or a decrease from baseline of at least 2 points. Higher scores indicate a worse outcome. Title: PHYSICIAN GLOBAL ASSESSMENT Minimum value: 0 Maximum value: 5
次要结局
- Skin assessment every visit(through study completion, 52 weeks)
- Skin pictures at baseline to 52 weeks(through study completion, 52 weeks)
- Patient Global Assessment (PtGA)(through study completion, 52 weeks)
- Dermatology Life Quality Index (DLQI)(through study completion, 52 weeks)
- Treatment Emergent Adverse Event (TEAEs)(through study completion, 52 weeks)
研究者
David Rosmarin
Chair, Department of Dermatology
Indiana University