Primary Hyperoxaluria Type 1Phase 2PHYOX7NCT04580420
Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
A Phase 2 study testing DCR-PHXC in people with Primary Hyperoxaluria Type 1 and End Stage Renal Disease, run by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company at 18 sites in 10 countries.
Trial summary
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Hyperoxaluria Type 1, End Stage Renal Disease
- What is being tested?
- DCR-PHXC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- 18 sitesin 10 countries
- Study dates
- 15 Apr 2021to 30 Jan 2032
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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)
Who cannot join
- 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
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- 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
Trial locations and contacts
18 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
18 sites in 10 countries
United States4 sites, 1 recruiting
- Active, not recruiting
Clinical Trial Site
Boston, Massachusetts, 02115
- Active, not recruiting
Clinical Trial Site
New York, New York, 10016
- Recruiting
Clinical Trial Site
Rochester, Minnesota, 55905
Clinical Trial Coordinator
- Active, not recruiting
Clinical Trial Site
San Francisco, California, 94143
France2 sites
- Withdrawn
Clinical Trial Site
Bron, 69677
- Withdrawn
Clinical Trial Site
Paris, 75019
Germany2 sites, 1 recruiting
- Recruiting
Clinical Trial Site
Bonn, 53127
Clinical Trial Coordinator
- Withdrawn
Clinical Trial Site
Heidelberg, 69120
Romania2 sites
- Withdrawn
Clinical Trial Site
Oradea, 410469
- Withdrawn
Clinical Trial Site
Oradea, 410562
Spain2 sites, 1 recruiting
- Recruiting
Clinical Trial Site
Barcelona, 08035
Clinical Trial Coordinator
- Withdrawn
Clinical Trial Site
Santa Cruz de Tenerife, 38320
United Kingdom2 sites, 1 recruiting
- Withdrawn
Clinical Trial Site
London, NWG 2Q3
- Recruiting
Clinical Trial Site
London, WC1N3JH
Clinical Trial Coordinatoor
Lebanon1 site, 1 recruiting
- Recruiting
Clinical Trial Site
Beirut, 00001
Clinical Trial Coordinatoor
United Arab Emirates1 site, 1 recruiting
- Recruiting
Clinical Trial Site
Dubai, +971
Clinical Trial Coordinator
About this study
Official title: 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
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Read the 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.
- Being tested
- DCR-PHXC
- Keywords
- PH1, ESRD
- Medical subject headings
- Primary hyperoxaluria type 1, Kidney Failure, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DCR-PHXCDrug
Also called Nedosiran
Monthly dosing throughout study period
| Study group | Role | What they receive |
|---|---|---|
| Open-Label DCR-PHXC | Experimental | Open-Label monthly subcutaneous injection of DCR-PHXC based on age and weight. Receives: Drug: DCR-PHXC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Incidence of Events
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.
Measured: 180 days
Safety: Incidence of Events
Characterize the safety of DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Measured: 180 days
Secondary outcomes (8)
Change from Baseline in Plasma Oxalate Concentration
To evaluate the effect of DCR-PHXC on Plasma Oxalate concentration from Baseline to day 180
Measured: 180 Days
To characterize the PK of DCR PHXC in patients with PH by observing secondary parameters of the area under the curve.
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including secondary parameters of area under the curve (AUC)
Measured: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing maximum observed concentration (Cmax).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including maximum observed concentration (Cmax)
Measured: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing minimum concentration (Cmin).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including minimum observed concentration (Cmin)
Measured: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing maximum concentration (Tmax).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including time to maximum concentration (Tmax)
Measured: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing terminal elimination half-life (t1/2).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including terminal elimination half-life (t1/2)
Measured: 180 days
Change in Frequency of Dialysis
Change from Baseline in the frequency of dialysis over 180 days
Measured: 180 days
Change in Duration of Dialysis
Change from Baseline in the duration of dialysis over 180 days
Measured: 180 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2020
- Study start15 Apr 2021
- Primary completion30 Dec 2031 (estimated)
- Study completion30 Jan 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Hyperoxaluria Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency (dept. 2834), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04580420
- Sponsor's protocol ID
- DCR-PHXC-204
- Acronym
- PHYOX7
- Status last verified
- Dec 2025
- First submitted
- 24 Sep 2020
- Last updated
- 24 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Cox JH, Boily MO, Caron A, Sheng T, Wu J, Ding J, Gaudreault S, Chong O, Surendradoss J, Gomez R, Lester J, Dumais V, Li X, Gumpena R, Hall MD, Waterson AG, Stott G, Flint AJ, Moore WJ, Lowther WT, Knight J, Percival MD, Tong V, Oballa R, Powell DA, King AJ. Characterization of CHK-336, A First-in-Class, Liver-Targeted, Small-Molecule Lactate Dehydrogenase Inhibitor for Hyperoxaluria Treatment. J Am Soc Nephrol. 2025 Apr 7;36(8):1535-1547. doi: 10.1681/ASN.0000000690.
PubMed 40193200