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Clinical Trial Summary

The DCR-PHXC-104 study is designed to assess the safety, tolerability, and pharmacological parameters of a single dose of DCR-PHXC in Primary Hyperoxaluria Type 3 (PH3). Participants should have had at least one stone event within 12 months of screening and intact renal function.

Clinical Trial Description

Potential participants are screened over an up-to-35-day period (with an extra 7-day period for participants who are required to repeat screening 24-hour urine collections or initially unanalyzable screening laboratory assessment samples) prior to randomization. Eligible participants will receive a single dose of DCR-PHXC or placebo on Day 1. In order to maintain the treatment blind, 24-hour urine oxalate (Uox) results that could unblind the study will not be reported to investigative sites or other blinded personnel until the study has been unblinded. It is expected that approximately 10 participants will be screened in order to randomize 6 participants (2:1 randomization; 4 nedosiran:2 placebo) to the study. Following the up-to-6-week screening period, participants will return to the clinic for interim visits up to Day 85. Visits occurring between the Day 1 and the Day 85 visit may be conducted as at-home telemedicine visits at the discretion of the Investigator. The total time on study for each participant is approximately 18 weeks. ;

Study Design

Related Conditions & MeSH terms

NCT number NCT04555486
Study type Interventional
Source Dicerna Pharmaceuticals, Inc.
Status Completed
Phase Phase 1
Start date September 14, 2020
Completion date September 7, 2021

See also
  Status Clinical Trial Phase
Active, not recruiting NCT04542590 - Natural History of Patients With PH3 and a History of Stone Events
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT05001269 - Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function Phase 2