An observational study in Primary Hyperoxaluria Type 3, sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company. Completed at 6 sites in 5 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2024-12-02.
Sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company (part of Novo Nordisk A/S) · Observational
This is a natural history study of adults, adolescents, and children (starting at birth) with genetically confirmed primary hyperoxaluria type 3 (PH3) who have a history of stone events during the last 3 years and/or the presence of pre existing stones detected by renal ultrasound at screening.
The relationship between the level of Uox and the incidence of kidney stones and/or nephrocalcinosis in patients with PH3 has not been established. The goal of this study is to record 24-hour Uox levels and the incidence of new stone formation and/or the degree of nephrocalcinosis in patients with PH3 over time.
Potential participants are screened over an up-to-7 week period according to the eligibility criteria and will then be followed every 6 months over an up-to-2-year period to determine the annualized new stone formation rate (number of new stones being formed per year) and the change in the degree of nephrocalcinosis (if applicable).
New stone formation is defined as occurrence of any of the following:
This is a non-interventional study that will last up to 2 years.
42 studies on the registry are indexed under Hyperoxaluria, Primary; 11 are open to participants now.
This study's enrollment of 7 is below the median of 730 across 13 observational studies indexed under Hyperoxaluria, Primary.
Browse Hyperoxaluria, Primary studies →Dicerna Pharmaceuticals, Inc., a Novo Nordisk company is the lead sponsor of 18 studies on the registry; 2 are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 4 (50%) have results posted.
First submitted before Dicerna Pharmaceuticals, Inc. became part of Novo Nordisk A/S.
Counted across the registry records on this site, refreshed daily.
This is a natural history study in adults, adolescents, and children (starting from birth) with genetically confirmed PH3 who have a history of stone events during the last 3 years and/or the presence of preexisting stones detected by renal ultrasounds at Screening, and an eGFR > 30 mL/min/1.73 m2 or serum creatinine below the 97th percentile for patients younger than 12 months old.
Key inclusion criteria
Uox ≥ 0.7 mmol/24 hours (adjusted per 1.73 m2 BSA in participants \< 18 years of age) OR if not able to collect 24-hour urine, average spot Uox to creatinine ratio at Screening above the 95th percentile for age:
Key Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Collect stone formation data in PH3 patients
Collect data on the rate of new stone formation in PH3 patients of at least 2 years of age
Time frame: Assess participants' stone formation rates over the course of 2 years
Assess relationship between urine oxalate (Uox) levels and stone formations in PH3 patients
Explore the potential relationship between Uox levels and new stone formation in patients (≥ 2 years of age) with genetically confirmed PH3 and relatively intact renal function
Time frame: Measure participants' Uox levels over the course of 2 years
Collect data on the degree of nephrocalcinosis in PH3 patients
Collect data on the degree of nephrocalcinosis in PH3 patients of at least 2 years of age
Time frame: Assess the change in nephrocalcinosis grade over the course of 2 years
This study is completed, as verified in Nov 2024. You cannot join it, but the record below documents what was studied.
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Dicerna Pharmaceuticals, Inc., a Novo Nordisk company →