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)
Who can participate
Age range12 Years
SexALL
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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
What they're measuring
1
The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs)
Timeframe: Through Day 29
Trial details
NCT IDNCT02795325
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company