临床试验/NCT05643872

NCT05643872招募中3 期

A Multicenter Open-Label Treatment Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita

Palvella Therapeutics, Inc.1 个研究点分布在 1 个国家目标入组 45 人开始时间: 2022年11月15日最近更新: 2022年12月9日

适应症

干预措施

试验速览

阶段: 3 期
状态: 招募中
发起方: Palvella Therapeutics, Inc.
入组人数: 45
试验地点: 1
主要终点: Treatment emergent adverse events

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS) trial.

The purpose of this OLT study is to investigate the safety of long term exposure and pharmacokinetics (PK) of QTORIN rapamycin 3.9% anhydrous gel or "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: 16 Years 至 —（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Completed the PALV-05 (VAPAUS) study
•Agree to contraceptive use

排除标准

•Females who are pregnant or breastfeeding
•Concomitant use of sirolimus or any sirolimus-containing medications (systemic or topical)
•Any significant concurrent condition that could adversely affect participation and/or the assessment of the safety and efficacy in the study
•Prior or planned treatment, including surgery or other medically necessary intervention deemed by the investigator that could adversely affect participation and/or the assessment of the safety and efficacy in the study

研究组 & 干预措施

PTX-022

Experimental

干预措施: PTX-022 (Drug)

结局指标

主要结局

Treatment emergent adverse events

时间窗: 6 months

次要结局

Pharmacokinetic parameters(Prior to dose, 12 hours and 24 hours)

研究者

发起方

Palvella Therapeutics, Inc.

申办方类型

Industry

责任方

Sponsor

研究点 (1)

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