Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD
Recruiting · 18 sites across 4 states
Always free
Study care at no cost to you
For your time and travel
Many studies pay you back
Most need no insurance or papers
Legal status usually isn't required; we'll tell you each study's requirements
Interpreters available
Ask for your language
Your choice
Voluntary — you can stop anytime
What is this study?
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
It is , overseen by an independent and licensed medical staff.
Read the full clinical description
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Who this study is looking for
In plain language, from the study's own rules. The study team confirms the full details with you — this isn't a final yes or no.
✅ You may be able to join if…
- •A person with PH1 (primary hyperoxaluria type 1) confirmed by genotyping (genetic testing).
- •Severe kidney impairment at screening, with estimated GFR (a kidney filtering measure) <30 mL/min normalized to 1.73 m² body-surface area (BSA).
- •Mean plasma oxalate level is >20 μmol/L during screening (plasma oxalate is a blood chemical related to PH1).
- •Can be on dialysis if hemodialysis or peritoneal dialysis total duration is <24 months and the dialysis plan was stable for at least 2 weeks before screening.
- •Must be able to sign informed consent (or have a parent/guardian consent if under 18) and follow study rules.
🚫 You may not be able to join if…
- •A history of prior liver transplant, or a planned liver transplant within 6 months of Day 1.
- •Evidence of severe systemic oxalosis, including major problems like certain organ calcifications (retina/heart/skin) or severe bone problems (severe bone pain, fractures, or bone deformities).
- •Any condition or comorbidity that could interfere with study compliance/data understanding or impact safety (examples include severe intercurrent illness or certain causes of active liver injury).
- •Use of an RNAi drug or other DCR-PHXC product within the last 6 months.
- •Certain prior serious reactions to oligonucleotide-based therapy (for example, severe thrombocytopenia, major liver injury by lab tests, severe flu-like symptoms, severe injection skin reactions, or clotting problems).
Are you a good fit?
Simplified highlights. The study team always confirms the full details with you.
- !Some conditions may not be a fit: Fatty liver disease (NASH/MASH)
What to expect, step by step
- 1
Usually a few weeks
The study team checks whether the study is a good fit for you, with a visit and sometimes lab tests. You can ask any questions before deciding.
- 2
Treatment
If you join and choose to continue, you receive the study treatment and are watched closely by medical staff.
- 3
Follow-up
After treatment, the team checks on your health and confirms the visit schedule with you. You can leave the study at any point.
Has this treatment been tested before?
Yes. This treatment has already been through earlier human studies for safety before reaching this stage.
What you need to know before you apply
What is this study testing?+
The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Is it safe? Who makes sure of that?+
This is a mid-stage study (Phase 2), looking at safety and whether the treatment works. Every study is reviewed and monitored by an independent ethics board (called an IRB) whose job is to protect participants, and care is overseen by licensed medical staff. You'll be told the known risks before you agree to anything, and you can stop at any time.
Will I get a placebo instead of the real treatment?+
Some studies compare a treatment against a placebo (an inactive version), and some don't. If this one does, the study team will explain your chances of receiving the active treatment before you decide. Nothing is hidden from you.
Do I have to stop taking my current medications?+
It depends on the study. Some let you stay on your current medications and some ask you to adjust them. Never stop a medication on your own — the study team will review everything with you first.
Does it cost anything? Will I be paid?+
The study treatment and study-related visits are provided at no cost to you. Some studies also pay for your time; the coordinator can tell you if this one does. You should never be asked to pay to take part.
Do I need insurance? Will anyone ask about my immigration status?+
No. You do not need health insurance to take part in a research study, and you will not be asked about your immigration status to join. Taking part is about whether you're a medical fit for the study.
What if English isn't my first language?+
You have the right to understand everything before you agree. Study sites can often provide materials or an interpreter in your language — you can ask the coordinator for one.
Is my information private?+
Yes. Your health information is only shared with the study sites you choose to be contacted by, and only to help match and enroll you. It is never sold, and you can ask us to delete it at any time.
Source: ClinicalTrials.gov · NCT04580420 · Locations: California · Massachusetts · Minnesota · New York