At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- DCR-PHXC (drug)
- How long the study runs
- Study runs about 129 months (dates as stated)
- About the drug or intervention
- DCR-PHXC — drug: Monthly dosing throughout study period
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is testing a medicine called DCR-PHXC for people with primary hyperoxaluria type 1 (PH1), a rare condition causing a build-up of oxalate, who also have end stage renal disease (ESRD), meaning their kidneys have stopped working properly. It looks at how safe the medicine is and how well it works. The sponsor is Dicerna Pharmaceuticals, Inc., a Novo Nordisk company.
Who can take part
- Right now, the study is only taking people under 6 years old, although four age groups (birth to under 2, 2 to 5, 6 to 11, and 12 and over) are planned overall
- A confirmed diagnosis of PH1, proven by genetic testing
- Kidney function (estimated glomerular filtration rate) below 30 mL/min
- A plasma oxalate level above 20 μmol/L, based on the average of two tests
- If on dialysis (haemodialysis or peritoneal dialysis), it must have been for less than 24 months and on a stable routine for at least 2 weeks before screening
- Not pregnant or breastfeeding, and agreement to use contraception during treatment and for at least 12 weeks after the last dose, for those who can have children
- Signed informed consent from the participant, or parent or legal guardian for children, with written assent from those aged 12 to under 18 where required
- Being covered by a health insurance system, where this applies under national rules
Who may not be able to
- A previous liver transplant, or a transplant planned within 6 months of the start (previous kidney transplant is allowed)
- Signs of severe oxalate build-up around the body, such as calcium deposits in the eyes, heart or skin, or a history of severe bone pain, broken bones or bone deformity
- Another condition that could affect taking part, safety, or understanding of results, such as serious illness, liver disease, other causes of kidney problems, or concerns about drug or heavy alcohol use
- Use of an RNA interference drug, including other DCR-PHXC, in the last 6 months
- A past severe reaction to oligonucleotide therapy, such as very low platelets, liver injury, severe flu-like symptoms, severe skin reaction at the injection site, or blood clotting problems
- Taking part in another study with an experimental medicine (other than DCR-PHXC) within 4 months before screening
- Liver blood test results above 1.5 times the normal limit
- A positive test for anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibodies at screening
- Known severe allergy to DCR-PHXC or any of its ingredients
- Being unable or unwilling to follow the study procedures and lifestyle requirements
What taking part involves
- • Receiving the study medicine DCR-PHXC
- • Not stated — ask the trial team
Time commitment: Not stated — ask the trial team about how long the study lasts, how many visits are needed, and what taking part involves.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- Not specified
- Who
- All
- Number of participants
- 28
- Started
- 2021-04-15
- Last checked
- 2025-12
Plain English Summary
What is this study?
- • Testing a new treatment for primary hyperoxaluria type 1
- • Phase2 - 28 participants
- • The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis
Who can take part?
- • Adults
- • Diagnosed with primary hyperoxaluria type 1
Where?
- • London - Clinical Trial Site
- • London - Clinical Trial Site
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
More detail
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: Not specified
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ been stable for at least 2 weeks
What the study is looking for
- ✓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
Who cannot take part
- ✗Prior liver transplantation; or scheduled transplantation within 6 months of Day 1. Prior kidney transplantation is...
- ✗Presence of any condition or other health conditions that would interfere with study compliance or data interpretation or...
- ✗Severe intercurrent illness
- ✗Known causes of active liver disease/injury (e.g., alcoholic liver disease, nonalcoholic fatty liver...
- ✗Non-PH related conditions contributing to kidney insufficiency
See the full criteria
Where Is This Study? (2 UK sites)
Clinical Trial Site
London NWG 2Q3, United Kingdom
Clinical Trial Site
London WC1N3JH, United Kingdom
How to Get in Touch
Novo Nordisk
Sponsor contactCONTACT
