Hyperoxaluria Treatment with DCR-PHXC – Phase II

Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection in Patients with Primary Hyperoxaluria

Protocol Description

In this study, researchers are evaluating a drug called DCR-PHXC, when given to children and adults with primary hyperoxaluria (PH) type 1 or type 2 (PH1 or PH2). More specifically, researchers will be studying how well the drug helps the liver to make less oxalate to reduce symptoms of PH. Lowering the levels of oxalate may have a positive effect on patients with PH because most of the symptoms from PH are due to the excessive amounts of oxalate accumulating in the body.

Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, this study is accepting participants, ages 6 and older who have been diagnosed with PH1 or PH2.
Males and Females: Ages 6 and older

Requirements

Participants will be randomly assigned to be given either the study drug or a placebo. The drug will be administered monthly by injection during study visits. There will be a total of 12 study visits for examinations, medical tests, and imaging.
Visits: 12
Duration: Up to 7 months

Status: Open to Enrollment

Source(s) of Support

Dicerna Pharmaceuticals

Primary Investigator

Michael Moritz, MD

Contact Information

For more information about the study or enrollment, please contact:
Vibha Chauhan, PhD, CCRP, CCRC
412-692-7924