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

• Status: Terminated
• Trial end date: 2016-10-14
• Target enrollment: 0
• Participant gender: All

Summary

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)

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dicerna Pharmaceuticals, Inc.

Criteria

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