A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)

This is an interventional treatment trial for Primary Hyperoxaluria Type 1 focused on measuring Primary Hyperoxaluria Type 1, PH1, Calcium Oxalate Stones, Kidney Stones, Liver Enzyme Deficiency, Genetic Diseases, Inborn Kidney Diseases, Metabolic Diseases, Inborn Errors, Urological Diseases, Carbohydrate Metabolism, Hyperoxaluria, AGT, RNAi, siRNA, DCR-PH1 Read More

Inclusion Criteria:

• Male or female, at least 12 years of age
• Diagnosis of PH1, confirmed by genotyping
• 24-hour urine oxalate excretion as defined in the protocol
• eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA
• Written informed consent for adults (≥18 years old, or per local regulatory requirement); written informed assent for adolescents (12 to <18 years old, or per local regulatory requirement)

Exclusion Criteria:

• Prior renal and/or hepatic transplantation
• Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment
• Pregnancy or lactation at the time of screening or enrollment
• Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
• Patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus or hepatitis C virus
• Moderate to severe liver impairment
• Liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) > 2 times upper limit of normal (ULN)
• History of severe reaction to a liposomal product or a known hypersensitivity to lipid products.
• Unable to collect required study samples or follow study procedures
• No clinically significant health concerns