A Natural History Study of Patients with Genetically Confirmed Primary Hyperoxaluria Type 3 with a History of Stone Events
- Conditions
- Primary Hyperoxaluria type 3N/A
- Registration Number
- LBCTR2021054742
- Lead Sponsor
- Dicerna Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Pending
- Sex
- All
- Target Recruitment
- 1
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
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
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ame: 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 Outcome Measures
Name Time Method ame: new stone formation and degree of nephrocalcinosis;Timepoints: N/A;Measure: N/A