A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary… (NCT03847909) | Clinical Trial Compass
CompletedPhase 2
A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2
United States35 participantsStarted 2019-10-28
Plain-language summary
The purpose of this study is to evaluate the efficacy and safety of DCR-PHXC in Children and Adults with Primary Hyperoxaluria Type 1 (PH1) and Primary Hyperoxaluria Type 2 (PH2)
Who can participate
Age range6 Years
SexALL
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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
What they're measuring
1
AUC From Day 90 To Day 180, Based on Percent Change From Baseline in 24-Hour Uox