NCT05180708已完成3 期
A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 87
- 试验地点
- 8
- 主要终点
- Patient Global Assessment of Activities Difficulty Scale
研究概览
简要总结
This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.
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
- 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 (16 years or older in the UK)
- •Diagnosed with Pachyonychia Congenita (PC), genetically confirmed
排除标准
- •Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications.
- •Any significant concurrent condition (including conditions involving or inferior to the ankle) that could adversely affect the patient's participation and/or the assessment of the safety and efficacy in the study.
- •Participation in any other interventional clinical research trial within 30 days prior to the screening visit or during the patient's participation in this study
研究组 & 干预措施
Active
Active Comparator
干预措施: QTORIN 3.9% rapamycin anhydrous gel (Drug)
Vehicle
Placebo Comparator
干预措施: Vehicle (Drug)
结局指标
主要结局
Patient Global Assessment of Activities Difficulty Scale
时间窗: 6 months
次要结局
- PROMIS physical function(6 months)
- Clinician global impression of severity (CGI-S)(6 months)
- Pain at its worst as assessed by numerical rating scale(6 months)
- Clinician global impression of change (CGI-C)(6 months)
- Patient global impression of severity (PGI-S)(6 months)
- Patient global impression of change (PGI-C)(6 months)
研究者
研究点 (8)
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