Hypophosphatasia - Activity Trackingin a Rare, Inherited Musculoskeletal Condition(HATRIC) - (HATRIC)

Klinische Studieneinheit König Ludwig Haus0 个研究点目标入组 21 人开始时间: 2023年6月21日最近更新: 2024年8月19日

适应症

试验速览

暂无简介。

•Confirmed clinical diagnosis of HPP based on one of the following:
•- ALP activity (age- and gender-adjusted) below lower limit of normal, measured at least two times within a minimum four-week interval
•- Genetic confirmation of the disease by documented variant within the ALPL Gene
•- Experience HPP-associated functional deficits requiring professional treatment (ie. Pain medication, physiotherapy)
•- Elevated substrates above upper limit of normal for PLP and/or urinary PEA
•- Symptoms of the disease

•- Inability to walk, or require the use of assistive devices for walking
•- Regular use of a wheelchair even for distances < 50m
•- Inability / unwillingness to comply with repeated testing requirements and wearing the
•activity tracking device
•- Currently enrolled in another clinical trial
•- Patient on enzyme replacement therapy with asfotase alfa

发起方

Klinische Studieneinheit König Ludwig Haus