Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
PHYOX7
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
1 other identifier
interventional
28
10 countries
18
Brief Summary
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Apr 2021
Longer than P75 for phase_2
18 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 24, 2020
CompletedFirst Posted
Study publicly available on registry
October 8, 2020
CompletedStudy Start
First participant enrolled
April 15, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 30, 2031
ExpectedStudy Completion
Last participant's last visit for all outcomes
January 30, 2032
December 24, 2025
December 1, 2025
10.7 years
September 24, 2020
December 23, 2025
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
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.
180 days
Safety: Incidence of Events
Characterize the safety of DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
180 days
Secondary Outcomes (8)
Change from Baseline in Plasma Oxalate Concentration
180 Days
To characterize the PK of DCR PHXC in patients with PH by observing secondary parameters of the area under the curve.
180 days
To characterize the PK of DCR PHXC in patients with PH by observing maximum observed concentration (Cmax).
180 days
To characterize the PK of DCR PHXC in patients with PH by observing minimum concentration (Cmin).
180 days
To characterize the PK of DCR PHXC in patients with PH by observing maximum concentration (Tmax).
180 days
- +3 more secondary outcomes
Other Outcomes (5)
Change from Baseline in the Short Form (36) Health Survey (SF-36®) in adults
180 days
Change from Baseline in the EQ-5D-5L™ in adults
180 days
Change from Baseline in the Pediatric Quality of Life Inventory (PedsQL™) in children
180 days
- +2 more other outcomes
Study Arms (1)
Open-Label DCR-PHXC
EXPERIMENTALOpen-Label monthly subcutaneous injection of DCR-PHXC based on age and weight.
Interventions
Eligibility Criteria
You may qualify if:
- 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).
- +6 more criteria
You may not qualify if:
- 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.
- +4 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (18)
Clinical Trial Site
San Francisco, California, 94143, United States
Clinical Trial Site
Boston, Massachusetts, 02115, United States
Clinical Trial Site
Rochester, Minnesota, 55905, United States
Clinical Trial Site
New York, New York, 10016, United States
Clinical Trial Site
Bron, 69677, France
Clinical Trial Site
Paris, 75019, France
Clinical Trial Site
Bonn, 53127, Germany
Clinical Trial Site
Heidelberg, 69120, Germany
Clinical Trial Site
Roma, 00165, Italy
Clinical Trial Site
Beirut, 00001, Lebanon
Clinical Trial Site
Casablanca, 2025, Morocco
Clinical Trial Site
Oradea, 410469, Romania
Clinical Trial Site
Oradea, 410562, Romania
Clinical Trial Site
Barcelona, 08035, Spain
Clinical Trial Site
Santa Cruz de Tenerife, 38320, Spain
Clinical Trial Site
Dubai, +971, United Arab Emirates
Clinical Trial Site
London, NWG 2Q3, United Kingdom
Clinical Trial Site
London, WC1N3JH, United Kingdom
Related Publications (1)
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.
PMID: 40193200DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Clinical Transparency (dept. 2834)
Novo Nordisk A/S
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 24, 2020
First Posted
October 8, 2020
Study Start
April 15, 2021
Primary Completion (Estimated)
December 30, 2031
Study Completion (Estimated)
January 30, 2032
Last Updated
December 24, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will not share