This is a double-blind, placebo-controlled, dose escalation trial of DCR-PHXC in Healthy Volunteers (HVs) and patients with Primary Hyperoxaluria (PH). Once safety has been established in HV, PH patients with a confirmed diagnosis of PH1 and PH2 will be enrolled across multiple dosing cohorts. The study design will allow enrollment of PH patient cohorts at a given dose level once safety has been demonstrated in HV at that dose level. The study will be conducted in two parts: Part A: Single ascending dose (SAD) in HV; Part B: SAD in patients with PH1 and PH2 (lagging Part A by 1 dose level cohort).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
43
DCR-PHXC is a novel, potent, and long-acting small interference ribonucleic acid (siRNA) molecule conjugated to N-acteylgalactosamine (GalNAc) that is designed to decrease liver oxalate production. DCR-PHXC is delivered via subcutaneous (SC) injection.
Single SC administration of placebo, which will be a sterile, preservative-free normal saline 0.9% solution for SC injection, which is of similar osmolality to the DCR-PHXC formulation.
Boston Children's Hospital
Boston, Massachusetts, United States
Centre d'Investigation Clinique - CIC 1407 - Hospices Civils de Lyon
Bron, France
Universitätsklinikum Bonn-Institut für Klinische Chemie und Klinische Pharmakologie
Bonn, Germany
University of Amsterdam
Amsterdam, Netherlands
Queen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
Birmingham Children's Hospital NHS Trust
Birmingham, United Kingdom
Clinical Trial Site
Wales, United Kingdom
Number of patients with Treatment-Related Adverse Events (TEAEs)
Time frame: Part A (SAD in HVs) screening through Day 29; Part B (SAD in PH patients) screening through Day 57
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