A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)

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