A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
Who can participate
Age range
12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Male or female, at least 12 years of age
* Diagnosis of PH1, confirmed by genotyping
* 24-hour urine oxalate excretion as defined in the protocol
* eGFR ≥ 40 mL/min normalized to 1.73 m2 BSA
* Written informed consent for adults (≥18 years old, or per local regulatory requirement); written informed assent for adolescents (12 to \<18 years old, or per local regulatory requirement)
Exclusion Criteria:
* Prior renal and/or hepatic transplantation
* Participation in any clinical study involving administration of any investigational drug within the 30 days before enrollment
* Pregnancy or lactation at the time of screening or enrollment
* Women of child-bearing potential must have a negative pregnancy test, cannot be breastfeeding and must be willing to use contraception
* Patients with a known history of human immunodeficiency virus (HIV) or active infection with hepatitis B virus or hepatitis C virus
* Moderate to severe liver impairment
* Liver function test abnormalities: alanine transaminases (ALT) and/or aspartate transaminases (AST) \> 2 times upper limit of normal (ULN)
* History of severe reaction to a liposomal product or a known hypersensitivity to lipid products.
* Unable to collect required study samples or follow study procedures
* No clinically significant health concerns
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The safety of DCR-PH1 evaluated by the proportion of subjects that experience adverse events (AEs)
Timeframe: Through Day 29
Trial details
NCT IDNCT02795325
SponsorDicerna Pharmaceuticals, Inc., a Novo Nordisk company