NCT03920228已完成2 期
A Multicenter, Four-Part, Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in the Treatment of Adults With Moderate to Severe Pachyonychia Congenita
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 73
- 试验地点
- 9
- 主要终点
- Patient Global Assessment of Activities Scale
研究概览
简要总结
This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.
This study includes four-parts, and if a participant completes all parts, the participant will have received at least 3-months of PTX-022 treatment.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
盲法说明
During the open-label period, there is no masking. During the randomized, double-blind, placebo-controlled period the participant and Investigator are masked.
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
- •Adult patients, 18 years or older
- •Diagnosed Pachyonychia Congenita (PC), genetically confirmed
- •Moderate to Severe PC
- •Able and willing to comply with all protocol-required activities
- •Willing and able to provide written informed consent
排除标准
- •Any significant concurrent condition (including involving the inferior to the ankle) that could adversely affect participation.
- •Any intentional changes in the patient's daily activities (associated with standing, walking and keeping balance), not resulting from an improvement in the patient's condition due to treatment.
- •Patient's deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, including adherence to study drug administration and other protocol-required activities.
研究组 & 干预措施
Open label period
Experimental
干预措施: PTX-022 (Drug)
Randomized period - Dosing A
Placebo Comparator
干预措施: Placebo (Drug)
Randomized period - Dosing B
Experimental
干预措施: PTX-022 (Drug)
Randomized period - Dosing B
Experimental
干预措施: Placebo (Drug)
Randomized period - Dosing C
Experimental
干预措施: PTX-022 (Drug)
结局指标
主要结局
Patient Global Assessment of Activities Scale
时间窗: 6 months
次要结局
- Clinician Global Impression of Change Scale(6 months)
- Pain at its worst as assessed by numerical rating scale(6 months)
- Number of steps taken as assessed by activity monitor(6 months)
研究者
研究点 (9)
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