ClinicalTrials.gov record NCT04580420
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
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This open-label Phase 2 study gives repeat monthly subcutaneous doses of DCR-PHXC to people with primary hyperoxaluria type 1 (PH1) and severe renal impairment, with or without dialysis. Participants attend monthly dosing visits through Day 180; those who complete Day 180 may continue open-label treatment and follow-up for up to 5 additional years, or until DCR-PHXC becomes commercially available, whichever comes first. Some participants or caregivers may be trained to give injections at home or receive home health support.
What it is studying. The study is testing the safety of monthly DCR-PHXC injections, how the drug behaves in the body (pharmacokinetics), its effect on blood (plasma) oxalate levels, and any changes in dialysis frequency or duration in people with PH1 and severe renal impairment.
What the researchers hope to learn. To characterize the safety of DCR-PHXC, its effect on plasma oxalate concentration, pharmacokinetic parameters (including AUC, Cmax, Cmin, Tmax, t1/2), and any changes in dialysis frequency or duration over 180 days.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- DCR-PHXC
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Note: Currently, the DCR-PHXC-204 study is only enrolling potential participants under 6 years of age.
- Four age groups of participants will be enrolled:
- adults and adolescents (aged ≥ 12 years)
- children 6 to 11 years of age
- children 2 to 5 years of age
- infants and newborns from birth to < 2 years of age
- . Documented diagnosis of PH1, confirmed by genotyping
- Estimated GFR at Screening <30mL/min normalized to 1.73m\^2 BSA
- Mean of 2 Plasma Oxalate >20μmol/L during screening
- 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.
- Male or Female
- Male participants:
- 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.
- 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:
- Not a woman of childbearing potential (WOCBP).
- 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.
- Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
- 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.
- Adolescents (12 to < 18 years of age, or older than 12 years but younger than the age of majority according to local regulations) must be able to provide written assent for participation.
- For children younger than 12 years of age, assent will be based on local regulations
- Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations)
Exclusion criteria
- Prior hepatic transplantation; or scheduled transplantation within 6 months of Day 1. Prior renal transplantation is allowed.
- 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)
- 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:
- Severe intercurrent illness
- Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver disease/steatohepatitis)
- Non-PH related conditions contributing to renal insufficiency
- 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)
- Use of an RNAi drug, other DCR-PHXC, within the last 6 months
- History of one or more of the following reactions to an oligonucleotide-based therapy:
- Severe thrombocytopenia (platelet count ≤ 100,000/µL)
- Hepatotoxicity, defined as alanine transaminase (ALT) or aspartate transaminase (AST) > 3 times the upper limit of normal (ULN) and total bilirubin > 2 × ULN or international normalized ratio (INR) >1.5
- Severe flu-like symptoms leading to discontinuation of therapy
- Localized skin reaction from the injection (graded severe) leading to discontinuation of therapy
- Coagulopathy/clinically significant prolongation of clotting time
- Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months before Screening.
- Liver function test abnormalities: ALT and/or AST >1.5 × ULN for age and gender
- Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening
- Known hypersensitivity to DCR-PHXC or any of its ingredients
- Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After up to a 35‑day screening period, participants attend monthly clinic visits to receive subcutaneous DCR‑PHXC injections through Day 180. They will have blood samples taken for plasma oxalate and drug level (pharmacokinetic) testing and undergo safety monitoring; dialysis frequency and duration will be recorded for those on dialysis. Participants who complete Day 180 may continue open‑label treatment and follow‑up for up to an additional 5 years with clinic visits every three months; participants or caregivers may be trained to give injections at home or a home health nurse may assist. Participation requires giving informed consent/assent and following the study procedures.
How the study is structured. Open-label, single-group Phase 2 drug study. Participants receive monthly subcutaneous injections of DCR-PHXC and are followed for safety, blood oxalate measurements, and drug level testing through Day 180. Participants who complete Day 180 may enter an extended open-label treatment period with clinic visits every three months for up to five more years. There is no placebo or blinding.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in France, Germany, Italy, Lebanon, Morocco, Romania, Spain, United Arab Emirates, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on primary hyperoxaluria
Important dates
- Study start
- Apr 15, 2021
- Primary completion
- Dec 30, 2031 (estimated)
- Study completion
- Jan 30, 2032 (estimated)
- Target enrollment
- 28 participants (estimated)
- Last updated on registry
- Dec 24, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Novo Nordisk 📞 (+1) 866-867-7178 ✉ clinicaltrials@novonordisk.com
Participating sites (18)
- Clinical Trial SiteActive Not Recruiting
San Francisco, California, 94143 · United States
- Clinical Trial SiteActive Not Recruiting
Boston, Massachusetts, 02115 · United States
- Clinical Trial SiteRecruiting
Rochester, Minnesota, 55905 · United States
- Clinical Trial Coordinator
- Clinical Trial SiteActive Not Recruiting
New York, New York, 10016 · United States
- Clinical Trial SiteWithdrawn
Bron, 69677 · France
- Clinical Trial SiteWithdrawn
Paris, 75019 · France
- Clinical Trial SiteRecruiting
Bonn, 53127 · Germany
- Clinical Trial Coordinator
- Clinical Trial SiteWithdrawn
Heidelberg, 69120 · Germany
- Clinical Trial SiteWithdrawn
Roma, 00165 · Italy
- Clinical Trial SiteRecruiting
Beirut, 00001 · Lebanon
- Clinical Trial Coordinatoor
Terms used on this page · explanations by MedicalUpdates.ai
- GFR
- A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
- at Screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- legal guardian
- A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- assent
- A child's or dependent adult's agreement to take part, given alongside the formal consent of a parent or legal representative.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- investigational
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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