NCT03392896

Study of DCR-PHXC-101 in Normal Healthy Volunteers and Patients With Primary Hyperoxaluria

A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Normal Healthy Volunteers and Open-Label Multi-Center Study in Patients With Primary Hyperoxaluria to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection (Subcutaneous Use)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Accepted

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).

Conditions

• Primary Hyperoxaluria

Interventions

Type	Name	Description
DRUG	DCR-PHXC	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.
DRUG	Placebo	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.

Timeline

• Start date: 2017-12-06
• Primary completion: 2019-11-19
• Completion: 2019-11-19
• First posted: 2018-01-08
• Last updated: 2024-09-19

Locations

7 sites across 5 countries: United States, France, Germany, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study