NCT04580420

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 28 (estimated)
• Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.

Detailed description

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.

Conditions

• Primary Hyperoxaluria Type 1
• End Stage Renal Disease

Interventions

Type	Name	Description
DRUG	DCR-PHXC	Monthly dosing throughout study period

Timeline

• Start date: 2021-04-15
• Primary completion: 2031-12-30
• Completion: 2032-01-30
• First posted: 2020-10-08
• Last updated: 2025-12-24

Locations

18 sites across 10 countries: United States, France, Germany, Italy, Lebanon, Morocco, Romania, Spain, United Arab Emirates, United Kingdom

Regulatory

• FDA-regulated drug study