RecruitingPhase 2NCT04580420

Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD

A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 and Severe Renal Impairment, With or Without Dialysis


Sponsor

Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Enrollment

28 participants

Start Date

Apr 15, 2021

Study Type

INTERVENTIONAL

Conditions

Summary

The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.


Eligibility

Plain Language Summary

Simplified for easier understanding

This study tests a drug called DCR-PHXC (also known as nedosiran) in people with a rare inherited kidney disease called Primary Hyperoxaluria Type 1 (PH1) who have severe kidney failure (end-stage renal disease, ESRD). PH1 causes the body to overproduce oxalate, which builds up and damages the kidneys. **You may be eligible if...** - You are currently under 6 years of age (note: only this age group is currently enrolling) - You have a confirmed genetic diagnosis of PH1 - Your kidney function is severely reduced (estimated GFR below 30 mL/min) - Your blood oxalate levels are elevated - If you are on dialysis, you have been on it for less than 24 months and your dialysis schedule has been stable **You may NOT be eligible if...** - You have normal or mildly reduced kidney function - You are pregnant or breastfeeding - You are not willing to use contraception as required by the study Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGDCR-PHXC

Monthly dosing throughout study period


Locations(18)

Clinical Trial Site

San Francisco, California, United States

Clinical Trial Site

Boston, Massachusetts, United States

Clinical Trial Site

Rochester, Minnesota, United States

Clinical Trial Site

New York, New York, United States

Clinical Trial Site

Bron, France

Clinical Trial Site

Paris, France

Clinical Trial Site

Bonn, Germany

Clinical Trial Site

Heidelberg, Germany

Clinical Trial Site

Roma, Italy

Clinical Trial Site

Beirut, Lebanon

Clinical Trial Site

Casablanca, Morocco

Clinical Trial Site

Oradea, Romania

Clinical Trial Site

Oradea, Romania

Clinical Trial Site

Barcelona, Spain

Clinical Trial Site

Santa Cruz de Tenerife, Spain

Clinical Trial Site

Dubai, United Arab Emirates

Clinical Trial Site

London, United Kingdom

Clinical Trial Site

London, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT04580420


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