Study population
liver function abnormalities, hypersensitivity
EVENT, HYPERSENSITIVITY
Key eligibility criteria for enrollment were a documented PH3 diagnosis confirmed by genotyping, age ≥ 6 years at time of informed consent, a history of ≥ 1-stone event within the last 12 months, 24-h Uox excretion ≥ 0.7 mmol (adjusted per 1.73 mKey exclusion criteria included prior or scheduled kidney or liver transplantation, dialysis, or any condition/comorbidity that would affect the study or patient safety. Individuals with a Pox level > 30 μmol/L, liver function abnormalities, hepatitis-/HIV-positive, and/or anti-dsDNA-positive were ineligible. We also excluded individuals who had used an RNAi drug within the last 6 months, a history of reactions to oligonucleotide-based therapy (or hypersensitivity to nedosiran), or received an investigational drug in a clinical study within 4 months (or five times the drug’s half-life) of PHYOX4.