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DCR-PHXC for Primary Hyperoxaluria Type 1 and End-Stage Renal Disease

This study is testing a drug called DCR-PHXC in people who have Primary Hyperoxaluria Type 1 (PH1), a rare genetic disorder that causes too much oxalate to build up in the body, and End-Stage Renal Disease (ESRD), which means their kidneys have failed. The main goal is to see how safe DCR-PHXC is over 180 days. The study is also looking at how well DCR-PHXC works. It is currently enrolling children under 6 years old, but plans to include adults, adolescents (12 years and older), and children from birth to 11 years old. Participants will receive monthly doses of DCR-PHXC.

Study design
This is an open-label (meaning everyone knows what treatment they are getting), repeat-dose, Phase 2 study. It plans to enroll 28 participants.
What's involved
You would have a screening period of up to 35 days, then monthly visits for DCR-PHXC dosing for 180 days. After that, you may continue receiving DCR-PHXC for up to 5 years with visits every 3 months, possibly at home.
Compensation
Not stated in the trial record.
Follow-up
Participants who complete the initial 180-day period may continue in an extended follow-up period for an additional 5 years, or until DCR-PHXC is commercially available.

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NCT04580420

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

Recruiting
PHASE2All AgesInterventionalTreatment
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
~28 participants
Updated 2025-12-24 on ClinicalTrials.gov
What's tested:DCR-PHXC

At a glance

Recruiting sites
6 of 18 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety: Incidence of Events
Measured over 180 days
+1 more outcome measured

Conditions

Primary Hyperoxaluria Type 1
End Stage Renal Disease

Explore related trials

Chronic Kidney Disease, Stage 5Primary Hyperoxaluria Type IAGXT wt Allele

Where it's being run

18 sites across 13 states
France2
Germany2
Romania2
Spain2
United Kingdom2
California1
Massachusetts1
Minnesota1

Study leadership

  • Clinical Transparency (dept. 2834) · STUDY_DIRECTOR · Novo Nordisk A/S

Who to contact

Novo Nordisk
[email protected]
(+1) 866-867-7178
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

A male participant with a female partner of childbearing potential must agree to use contraception during the treatment period and for at least 12 weeks after the last dose of study intervention and refrain from donating sperm during this period. 2. Female participants:
A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
OR
A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 12 weeks after the last dose of study intervention and agrees to refrain from harvesting/freezing eggs during this period. 3. Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. 7. Participant (and/or participant's parent or legal guardian if participant is a minor \[defined as patient \<18 years of age, or younger than the age of majority according to local regulations\]) is capable of giving signed informed consent, which includes compliance with the requirement and restrictions listed in the informed consent form (ICF) and in the protocol.

What this trial measures

  • Safety: Incidence of Events180 days

    To assess the efficacy of DCR-PHXC in lowering Pox in participants with PH1 and severe renal impairment, with or without hemodialysis or peritoneal dialysis.

  • Safety: Incidence of Events180 days

    Characterize the safety of DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.