Actively Recruiting
Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
Led by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Updated on 2025-12-24
28
Participants Needed
18
Research Sites
563 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
CONDITIONS
Official Title
Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be younger than 6 years of age
- Participants will be enrolled in one of four age groups: adults and adolescents (12 years and older), children aged 6 to 11 years, children aged 2 to 5 years, and infants or newborns from birth to less than 2 years
- Confirmed diagnosis of Primary Hyperoxaluria Type 1 by genetic testing
- Estimated kidney function (GFR) below 30 mL/min per 1.73 m² at screening
- Average of two plasma oxalate measurements above 20 micromol/L during screening
- For dialysis patients, total dialysis duration must be less than 24 months and stable for at least 2 weeks before screening
- Both male and female participants are eligible
- Male participants with female partners of childbearing potential must agree to use contraception during treatment and for 12 weeks after last dose, and avoid sperm donation during this time
- Female participants must not be pregnant or breastfeeding and either not be of childbearing potential or agree to use contraception during treatment and for 12 weeks after last dose
- Participants or their legal guardians must be able to give informed consent and comply with study requirements
- Adolescents aged 12 to under 18 years must provide written assent
- Children under 12 years assent will follow local regulations
- Participants must be affiliated with or beneficiaries of a health insurance system if required by local law
You will not qualify if you...
- Previous liver transplant or scheduled liver transplant within 6 months of Day 1 (kidney transplant allowed)
- Evidence of severe systemic oxalosis, including retinal, heart, or skin calcifications, severe bone pain, fractures, or bone deformities
- Conditions or illnesses that could interfere with study compliance or safety, such as severe illness, active liver disease, or non-PH causes of kidney failure
- History of drug abuse or excessive alcohol intake in the last 2 years
- Use of RNA interference drugs or DCR-PHXC within the past 6 months
- History of serious reactions to oligonucleotide therapies, including severe thrombocytopenia, liver toxicity, severe flu-like symptoms, severe skin reactions, or blood clotting problems
- Participation in other clinical trials with investigational drugs (except DCR-PHXC) within 4 months before screening
- Abnormal liver function tests exceeding 1.5 times the upper normal limit for age and gender
- Positive test for anti-double-stranded DNA antibodies at screening
- Known allergy to DCR-PHXC or its ingredients
- Inability or unwillingness to follow study procedures and lifestyle guidelines
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 18 locations
1
Clinical Trial Site
San Francisco, California, United States, 94143
Active, Not Recruiting
2
Clinical Trial Site
Boston, Massachusetts, United States, 02115
Active, Not Recruiting
3
Clinical Trial Site
Rochester, Minnesota, United States, 55905
Actively Recruiting
4
Clinical Trial Site
New York, New York, United States, 10016
Active, Not Recruiting
5
Clinical Trial Site
Bron, France, 69677
Withdrawn
6
Clinical Trial Site
Paris, France, 75019
Withdrawn
7
Clinical Trial Site
Bonn, Germany, 53127
Actively Recruiting
8
Clinical Trial Site
Heidelberg, Germany, 69120
Withdrawn
9
Clinical Trial Site
Roma, Italy, 00165
Withdrawn
10
Clinical Trial Site
Beirut, Lebanon, 00001
Actively Recruiting
11
Clinical Trial Site
Casablanca, Morocco, 2025
Withdrawn
12
Clinical Trial Site
Oradea, Romania, 410469
Withdrawn
13
Clinical Trial Site
Oradea, Romania, 410562
Withdrawn
14
Clinical Trial Site
Barcelona, Spain, 08035
Actively Recruiting
15
Clinical Trial Site
Santa Cruz de Tenerife, Spain, 38320
Withdrawn
16
Clinical Trial Site
Dubai, United Arab Emirates, +971
Actively Recruiting
17
Clinical Trial Site
London, United Kingdom, NWG 2Q3
Withdrawn
18
Clinical Trial Site
London, United Kingdom, WC1N3JH
Actively Recruiting
Research Team
N
Novo Nordisk
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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