Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD (PHYOX7)
Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD (PHYOX7)
ClinicalTrials.gov ID: NCT04580420
Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Information provided by: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (Responsible Party)
Last Update Posted: 2024-03-28
Brief Summary:
The aim of this study is to evaluate DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without dialysis.
Detailed Description:
This is an open-label, repeat-dose, Phase 2 study of DCR-PHXC in participants with PH1 or PH2 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 3 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 2 years from first participant, first visit, to last participant, last Day 180 visit, with up to an additional 3 years of extended follow-up.
Official Title:
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
Intervention / Treatment:
- Drug: DCR-PHXC
| Category | Value |
|---|---|
| Study Start (Actual) |
2021-04-15
|
| Primary Completion (Estimated) |
2024-11
|
| Study Completion (Estimated) |
2025-05
|
| Enrollment (Estimated) | 17 |
| Study Type | Interventional |
| Phase | Phase 2 |
| Other Study ID Numbers |
DCR-PHXC-204
|