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

NCT03392896 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 43

Last updated 2024-09-19

No results posted yet for this study

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

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.

Sponsors & Collaborators

  • Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2017-12-06
Primary Completion
2019-11-19
Completion
2019-11-19
FDA Drug
Yes

Countries

  • United States
  • France
  • Germany
  • Netherlands
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03392896 on ClinicalTrials.gov