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
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Summary
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
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 partic…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- No
Note: Currently, the DCR-PHXC-204 study is only enrolling potential participants under 6 years of age. Inclusion Criteria: 1. Four age groups of participants will be enrolled: 1. adults and adolescents (aged ≥ 12 years) 2. children 6 to 11 years of age 3. children 2 to 5 years of age 4. infants and newborns from birth to \< 2 years of age 2. . Documented diagnosis of PH1, confirmed by genotyping 3. Estimated GFR at Screening \<30mL/min normalized to 1.73m\^2 BSA 4. Mean of 2 Plasma Oxalate \>20μmol/L during screening 5. For participants receiving hemodialysis or peritoneal dialy…
Interventions
- DrugDCR-PHXC
Monthly dosing throughout study period
Locations (18)
- Clinical Trial SiteSan Francisco, California
- Clinical Trial SiteBoston, Massachusetts
- Clinical Trial SiteRochester, Minnesota
- Clinical Trial SiteNew York, New York
- Clinical Trial SiteBron
- Clinical Trial SiteParis