VALO-2: A Multicenter, Phase 3b, Open-Label Treatment Extension Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 36
- 试验地点
- 4
- 主要终点
- Incidence of Treatment-Emergent Adverse Events
研究概览
简要总结
VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study. The purpose of the OLE study is to investigate long term exposure to investigational PTX-022 and evaluate safety and efficacy data. A sub-study is included to evaluate safety and efficacy of patients with genotyped keratin mutations of KRT6C and KRT17 not previously treated with investigational PTX-022.
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
- •Adult patients 18 years or older,
- •Previously completed the VALO protocol, or VALO-2 K6C/17 sub-study, and received meaningful benefit from investigational PTX-022 as determined by the clinician.
排除标准
- •Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022
研究组 & 干预措施
Open label treatment arm
PTX-022 QTORIN
干预措施: PTX-022 (Drug)
结局指标
主要结局
Incidence of Treatment-Emergent Adverse Events
时间窗: 18 Months
Changes in vital sign measurements from baseline
时间窗: 18 months
Changes in weight measurements from baseline
时间窗: 18 months
Changes in clinical laboratory measurements from baseline
时间窗: 18 months
次要结局
- Pain at its worst as assessed by NRS(18 months)
- PROMIS Physical Function as assessed by likert scale(18 months)
- Clinician Global Impression of Severity as assessed by likert scale(18 months)
- Patient Global Assessment of Activities Scale(18 Months)
- PROMIS Pain Interference Short 6A as assessed by likert scale(18 months)
- Patient Global Impression of Severity as assessed by likert scale(18 months)