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Looking for participantsPhase2

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

Sponsor: Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

NCT ID: NCT04580420

View on ClinicalTrials.gov ↗

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.

Primary Hyperoxaluria Type 1End Stage Renal Disease

How this trial compares with your answers

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What we know so far

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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
Note: Currently, the DCR-PHXC-204 study is only enrolling potential participants under 6 years of age. Inclusion Criteria: 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. 6. Male or Female 1. 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. 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: 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. 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. 1. 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. 2. For children younger than 12 years of age, assent will be based on local regulations 8. Affiliated with or is a beneficiary of a health insurance system (if applicable per national regulations) 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) 2. 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 3. Severe flu-like symptoms leading to discontinuation of therapy 4. Localized skin reaction from the injection (graded severe) leading to discontinuation of therapy 5. Coagulopathy/clinically significant prolongation of clotting time 6. Participation in any clinical study in which they received an investigational medicinal product (IMP) other than DCR-PHXC within 4 months before Screening. 7. Liver function test abnormalities: ALT and/or AST \>1.5 × ULN for age and gender 8. Positive anti-double-stranded deoxyribonucleic acid (anti-dsDNA) antibody test at Screening 9. Known hypersensitivity to DCR-PHXC or any of its ingredients 10. Inability or unwillingness to comply with the specified study procedures, including the lifestyle considerations

Where Is This Study? (2 UK sites)

Clinical Trial Site

London NWG 2Q3, United Kingdom

WITHDRAWN

Clinical Trial Site

London WC1N3JH, United Kingdom

Recruiting

How to Get in Touch

Novo Nordisk

Sponsor contact

CONTACT

(+1) 866-867-7178 clinicaltrials@novonordisk.com
Data sourced from ClinicalTrials.gov · Last verified: 2025-12