A Phase 1 Placebo-Controlled Double-Blind Multicenter Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamics of a Single Dose of DCR-PHXC in Patients with Primary Hyperoxaluria Type 3

Brief description of study

This study is being run to test a new drug for use in people with primary hyperoxaluria type 3 (PH3). The study drug is called DCR-PHXC and is administered using a needle that is placed under the skin (subcutaneous injection). The purpose of this study is to learn more about how DCR-PHXC works in the body, how well it helps the liver make less oxalate to reduce symptoms of PH in patients at least 6 years of age with PH3, and to learn if the study drug is safe to use for the treatment of PH3. The study drug, DCR-PHXC, has not been approved by the US Food and Drug Administration and is therefore an experimental treatment. In this study, DCR-PHXC will be compared to a placebo. A placebo is a treatment that looks exactly like the study drug but does not contain any active ingredient.


Clinical Study Identifier: s20-01124
ClinicalTrials.gov Identifier: NCT04555486
Principal Investigator: David S. Goldfarb.
Other Investigator: Lama Nazzal.


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