Genetic Liver-Targeted Diseases

Primary HYperoxaluria Observational Study (PHYOS)

Primary HYperoxaluria Observational Study (PHYOS)

PHYOS is an international, multicenter, observational study that is collecting data on key biochemical parameters implicated in the pathogenesis of PH1 to better understand the baseline disease state, knowledge that will help guide long-term drug development plans. The study’s primary objective is to measure changes in oxalate, glycolate and other metabolites over a six-month period in patients with PH1. PHYOS investigators are also collecting data on the clinical manifestations of PH1, fluid intake and quality of life.

Investigators are now enrolling patients for the PHYOS study, which will eventually include up to 50 participants at as many as 12 sites worldwide. The study population will consist of two cohorts: Cohort A is enrolling patients age 12 years or older, as individuals in this age group will be considered for upcoming trials of potential PH1 therapeutics. Cohort B will enroll patients aged 6 to 11.9 years, after determination that patients of this age group may be enrolled in PH1 clinical trials.