Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Age
Any age
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC.
OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH.
For participants rolling over from a multidose study of DCR-PHXC, enrollment should occur within a window of 25 to 75 days from the last dose of study intervention.
Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula in participants aged ≥ 18 years, or the multivariate equation by Schwartz in participants aged 12 months to 17 years. In Japan, the cystatin C-based Uemura formula will be used for participants aged 12 months to \<2 years, the creatinine-based Uemura formula by will be used for participants aged 2 to 17 years, and the equation by Matsuo will be used in participants aged ≥ 18 years.
You may not be if
Renal or hepatic transplantation (prior or planned within the study period)
Plasma oxalate \> 30 µmol/L
Currently on dialysis
Documented evidence of clinical manifestations of systemic oxalosis
