A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment With or Without Dialysis

Brief description of study

The purpose of this study is to learn whether the study drug DCR-PHXC is safe and able to treat patients with Primary Hyperoxaluria Type 1 or 2 (known as PH1 or PH2) who also have severe kidney impairment with or without dialysis. People with PH1 or PH2 are deficient in an enzyme (protein) responsible for preventing the accumulation of a substance called oxalate. The recurrent buildup of oxalate stones in the kidneys and other organs (such as the liver) leads to kidney and other organ failure. DCR-PHXC is an investigational drug that reduces the amount of oxalate produced by another protein. It is considered investigational because it is not Food and Drug Administration (FDA) approved to treat PH and is being tested in research studies like this one.

Clinical Study Identifier: s20-01481
ClinicalTrials.gov Identifier: NCT04580420
Principal Investigator: David S. Goldfarb.

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