Loading clinical trials...

Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD | Clinical Trials | Clareo Health

RECRUITINGPHASE2

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

NCT04580420•Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

View on ClinicalTrials.gov

Summary

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

Detailed Description

The DCR-PHXC-204 study is listed under study number NN7022-8398 in Novo Nordisk systems. This is an open-label, repeat-dose, Phase 2 study of DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis. Following the up-to-35- day screening period, participants will return to the clinic for monthly dosing visits through Day 180. Participants successfully completing the Day 180 visit will continue on to an extended follow-up period and receive open-label DCR-PHXC for an additional 5 years, or until DCR-PHXC is commercially available, whichever comes first. As participants in this extended treatment period will return to the clinic only every 3 months, participants and/or their caregivers may be trained in the at-home administration of DCR-PHXC or home health nurses may assist with administration of DCR -PHXC. The total duration of the study is approximately 5 years from first participant, first visit, to last participant, last Day 180 visit, with up to an additional 5 years of extended follow-up.

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Note: Currently, the DCR-PHXC-204 study is only enrolling potential participants under 6 years of age.
•1. Four age groups of participants will be enrolled:
•1. adults and adolescents (aged ≥ 12 years)
•2. children 6 to 11 years of age
•3. children 2 to 5 years of age
•4. infants and newborns from birth to \< 2 years of age
•2. . Documented diagnosis of PH1, confirmed by genotyping
•3. Estimated GFR at Screening \<30mL/min normalized to 1.73m\^2 BSA
•4. Mean of 2 Plasma Oxalate \>20μmol/L during screening
•5. For participants receiving hemodialysis or peritoneal dialysis total duration of hemodialysis or peritoneal dialysis must be less than 24 months and hemodialysis or peritoneal dialysis regimen must have been stable for at least 2 weeks prior to Screening.
+13 more criteria

Exclusion Criteria

•1. Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed.
•2. Documented evidence of clinical manifestations of severe systemic oxalosis (including preexisting retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
•3. Presence of any condition or comorbidities that would interfere with study compliance or data interpretation or potentially impact patient safety including, but not restricted to:
•1. Severe intercurrent illness
•2. Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis)
•3. Non-PH related conditions contributing to renal insufficiency
•4. Physician concerns about intake of drugs of abuse or excessive alcohol intake, or history of excessive alcohol intake in the 2 years prior to enrollment (defined as ≥ 21 units of alcohol per week in men and ≥ 14 units of alcohol per week in women; where a "unit" of alcohol is equivalent to a 12-ounce beer, 4-ounce glass of wine, or 1ounce shot of hard liquor)
•4. Use of an RNAi drug, other DCR-PHXC, within the last 6 months
•5. History of one or more of the following reactions to an oligonucleotide-based therapy:
•1. Severe thrombocytopenia (platelet count ≤ 100,000/µL)
+9 more criteria

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

Key Dates

Start Date

April 15, 2021

Primary Completion Date

December 30, 2031

Completion Date

January 30, 2032

Last Updated

December 24, 2025

Enrollment

ESTIMATED participants

Conditions

Primary Hyperoxaluria Type 1End Stage Renal Disease

Interventions

DCR-PHXC

DRUG

Contacts

Novo Nordisk

CONTACT

(+1) 866-867-7178 clinicaltrials@novonordisk.com

EndoForce Post Approval Study

NCT07146854

End Stage Renal Disease (ESRD)

View study

RECRUITINGNA

Blood Pressure Slopes and Ultrafiltration in Hemodialysis Patients

NCT04163614

End Stage Renal DiseaseHypertension+2 more

View study

RECRUITINGNA

Maturation of Arteriovenous Fistula With Automated Sonography Assessments Trial

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: December 24, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

View ClinicalTrials.gov Terms and Conditions

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

Inclusion Criteria

Exclusion Criteria

EndoForce Post Approval Study

Blood Pressure Slopes and Ultrafiltration in Hemodialysis Patients

Maturation of Arteriovenous Fistula With Automated Sonography Assessments Trial

Comparing Surgical and Endovascular Arteriovenous Fistula Creation

Efficacy and Safety of XyloCore Peritoneal Dialysis Solution.

Xeltis Hemodialysis Access (aXess) Conduit: aXess-E Study