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This study evaluates the drug DCR-PHXC compared with a saline control in children and adults with primary hyperoxaluria type 1 and type 2. It included patients with a genetically confirmed diagnosis who met specific urinary oxalate requirements. It was conducted in Phase 2 and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Key Inclusion Criteria: * Capable and willing to provide written informed consent or assent * Documented diagnosis of PH1 or PH2, confirmed by genotyping * Must meet the 24 hour urine oxalate excretion requirements * Less than 20% variation between the two 24-hour urinary creatinine excretion values derived from the two 24-hour urine collections in the screening period * Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 BSA Key Exclusion Criteria: * Renal or hepatic transplantation (prior or planned within the study period) * Currently on dialysis or anticipated requirement for dialysis during the study period * Plasma oxalate \>30 µmol/L * Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations) * Use of an RNA interference (RNAi) drug within the last 6 months * Participation in any clinical study in which you received an investigational medicinal product (IMP) within 4 months before Screening * Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \>1.5 times upper limit of normal (ULN) for age and gender * Inability or unwillingness to comply with study procedures