Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- End Stage Renal Disease
- Primary Hyperoxaluria
- Primary Hyperoxaluria Type 2
- Type
- Interventional
- Phase
- Phase 2
- Design
- Allocation: N/AIntervention Model: Sequential AssignmentMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Younger than 125 years
- Gender
- Both males and females
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 successful...
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 will be trained in the at-home 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.
Tracking Information
- NCT #
- NCT04580420
- Collaborators
- Not Provided
- Investigators
- Study Chair: Alexandra Haagensen, MD, MBA Dicerna Pharmaceuticals, Inc.
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