Primary Hyperoxaluria type 3 N/A
Conditions
Interventions
Sponsors
Dicerna Pharmaceuticals, Inc.
Dicerna Pharmaceuticals INC.
Eligibility
Sex/Gender
All
Age
2 Years to 85 Years
Inclusion criteria
Inclusion criteria: Key inclusion criteria include • Genetically confirmed PH3 • History of stone events (defined as presence of calcifications in the urinary tract and/or kidney, their relative location, and the number and size of stones) during the last 3 years and/or presence of pre-existing stones detected by renal ultrasound at Screening • Uox > 0.7 mmol/24 hours normalized to 1.73 m2 BSA • eGFR at Screening = 30 mL/min • Able to accommodate 24-hour urine collection
Exclusion criteria
Exclusion criteria: Exclusion Criteria Participants are excluded from the study if any of the following criteria apply: Medical Conditions 1. Prior hepatic transplantation; or planned transplantation within the study period 2. Currently receiving dialysis or anticipating requirement for dialysis during the study period
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Name: The objective of this study is to collect data on stone formation and the degree of nephrocalcinosis in patients (= 2 years of age) with genetically confirmed PH3 and relatively intact renal function and to explore the potential relationship between Uox and new stone formation.;Timepoints: 4;Measure: stone formation | — |
Secondary
| Measure | Time frame |
|---|---|
| Name: new stone formation and degree of nephrocalcinosis;Timepoints: N/A;Measure: N/A | — |
Countries
Canada, France, Germany, Lebanon, Poland, United Kingdom, United States of America
Contacts
Public ContactSarah Kharsa
Clinart MEA
Outcome results
None listed