Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria
Status:
Completed
Trial end date:
2019-11-19
Target enrollment:
Participant gender:
Summary
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).