A Study to Determine the Prevalence and Clinical Characteristics Associated With PKD1 Gene Variant Groups in Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Vertex Pharmaceuticals Incorporated46 个研究点分布在 9 个国家目标入组 401 人开始时间: 2024年12月19日最近更新: 2026年9月9日

适应症

试验速览

阶段: 不适用
状态: 进行中（未招募）
发起方: Vertex Pharmaceuticals Incorporated
入组人数: 401
试验地点: 46
主要终点: Part A: Percentage of PKD1 Gene Variant Groups in the ADPKD Population

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

The purpose of this study is to estimate the prevalence, demographic, and clinical characteristics of PKD1/2 gene variant groups in the ADPKD population.

研究设计

研究类型: Observational
观察模型: Cohort
时间视角: Prospective

入排标准

年龄范围: 12 Years 至 65 Years（Child, Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Estimated glomerular filtration rate (eGFR) greater than or equal to (≥) 30 milliliter per minute (mL/min)/1.73 m^2
•Willing and able to comply with scheduled visits and other study procedures
•A pre-existing diagnosis of ADPKD as defined in the protocol

排除标准

•History of kidney disease other than ADPKD that in the opinion of the investigator would independently impact the natural history of ADPKD
•History of solid organ or bone marrow transplantation or nephrectomy
•Ongoing renal replacement therapy or planning to start renal replacement therapy less than or equal to (≤)12 months from the Genotyping Visit in Part A
•Other protocol defined Inclusion/Exclusion criteria will apply.

研究组 & 干预措施

Part B

Participants who have completed all assessments in Part A and have a subset of variants in PKD1 will complete 1 year of follow-up.

Part A

Eligible participants will complete a single visit to complete a blood sample collection for PKD1/2 genotyping.