Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD

The DCR-PHXC-204 study is listed under study number NN7022-8398 in Novo Nordisk systems.

This is an open-label, repeat-dose, Phase 2 study of DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis. Following the up-to-35- day screening period, participants will return to the clinic for monthly dosing visits through Day 180. Participants successfully completing the Day 180 visit will continue on to an extended follow-up period and receive open-label DCR-PHXC for an additional 5 years, or until DCR-PHXC is commercially available, whichever comes first. As participants in this extended treatment period will return to the clinic only every 3 months, participants and/or their caregivers may be trained in the at-home administration of DCR-PHXC or home health nurses may assist with administration of DCR -PHXC.

The total duration of the study is approximately 5 years from first participant, first visit, to last participant, last Day 180 visit, with up to an additional 5 years of extended follow-up.