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A Phase 2 Open-Label Multicenter Study to Evaluate the Efficacy,
Safety, and Pharmacokinetics of Nedosiran in Pediatric Patients from
Birth to 11 Years of Age with Primary Hyperoxaluria and Relatively
Intact Renal Function (PHYOX8)
Rochester, Minn.
The purpose of this study is to evaluate the safety, effectiveness, and pharmacokinetics (PK, how a drug is metabolized by the body) of Nedosiran in neonates, infants, and young children (birth to 11 years of age) with primary hyperoxaluria (type 1 [PH1] and type 2 [PH2]), and relatively intact renal function based upon eGFR and serum creatinine.
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Assessment of Health-related Quality of Life in Rare Kidney Stone Formers in the Rare Kidney Stone Consortium
Rochester, Minn.
Assessment of Health-related Quality of Life in Rare Kidney Stone Formers in the Rare Kidney Stone Consortium
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ILLUMINATE-B: An Open-Label Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
Rochester, Minn.
The purpose of the study is to determine if the investigational medicine, lumasiran, is able to reduce the production of oxalate in the liver and to evaluate the safety of lumasiran and how the body responds to it.
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