What is DCR-PHXC studying for Primary Hyperoxaluria Type 1?

DCR-PHXC is being studied to assess its safety and effects in people with Primary Hyperoxaluria Type 1 who have severe kidney impairment, including those on dialysis. The research aims to understand how the drug behaves in the body and its impact on important health markers over time. This study is sponsored by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company.

Who may be eligible for this Primary Hyperoxaluria Type 1 clinical trial?

Participants under 6 years old with a confirmed diagnosis of Primary Hyperoxaluria Type 1 and severe kidney impairment may be eligible. Both males and females can participate, provided they meet specific health and treatment stability criteria. Please review eligibility criteria for more details.

Is there any cost to participate in this Primary Hyperoxaluria Type 1 clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses to and from study visits may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 2

All Genders

ID04580420

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

Led by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company · Updated on 2025-12-24

Participants Needed

Research Sites

4 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating DCR-PHXC in individuals with Primary Hyperoxaluria Type 1 PH1 who have severe kidney impairment, including those on dialysis. This Phase 2 open-label study aims to assess the safety and effectiveness of DCR-PHXC in this specific patient group, which includes adults, children, and infants under 6 years old. The study is conducted by Dicerna Pharmaceuticals, Inc., a Novo Nordisk company, and focuses on participants with a confirmed PH1 diagnosis and significant kidney function decline. Participants will receive monthly subcutaneous injections of DCR-PHXC, with dosing schedules adjusted according to age and weight. After a screening period of up to 35 days, monthly dosing visits will continue through Day 180. Those completing the initial treatment phase may enter an extended follow-up period of up to five years or until the drug becomes commercially available. During this extension, dosing may be administered at home by participants, caregivers, or home health nurses with training. Throughout the study, participants will attend regular clinic visits for dosing and assessment. Researchers will monitor safety by tracking incidence of adverse events over 180 days. They will also evaluate changes in plasma oxalate levels, dialysis frequency and duration, and pharmacokinetics of DCR-PHXC. The total study duration can extend up to 10 years including the extended follow-up phase. Participants health and treatment compliance will be regularly assessed to understand the long-term effects of DCR-PHXC.

CONDITIONS

Brief Title

Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD

Who Can Participate

All Genders

Eligibility Criteria

You may qualify if you...

Participants under 6 years of age are currently enrolling.
Four age groups: adults/adolescents (≥12 years), children 6 to 11 years, children 2 to 5 years, infants/newborns from birth to <2 years.
Confirmed diagnosis of Primary Hyperoxaluria Type 1 by genetic testing.
Estimated glomerular filtration rate (GFR) below 30 mL/min/1.73m² at screening.
Mean plasma oxalate above 20 µmol/L during screening.
For dialysis patients, less than 24 months on hemodialysis or peritoneal dialysis with stable treatment for at least 2 weeks.
Male and female participants with contraception requirements as applicable.
Ability to provide informed consent or assent according to age and local regulations.
Affiliation with or beneficiary of a health insurance system if applicable.

You will not qualify if you...

Prior liver transplant or scheduled liver transplant within 6 months; prior kidney transplant allowed.
Severe systemic oxalosis symptoms such as retinal, heart, or skin calcifications, severe bone pain, fractures, or deformities.
Conditions interfering with study compliance or safety, including severe illness, active liver disease, non-PH kidney disease, or excessive alcohol or drug use.
Use of RNAi drugs or DCR-PHXC within 6 months.
History of severe reactions to oligonucleotide therapies, including low platelets, liver toxicity, severe flu-like symptoms, severe skin reactions, or clotting problems.
Participation in other investigational drug studies within 4 months before screening.
Elevated liver enzymes (ALT/AST) above 1.5 times normal for age and gender.
Positive anti-double-stranded DNA antibody test.
Known allergy to DCR-PHXC or its ingredients.
Inability or unwillingness to follow study procedures and lifestyle considerations.

Research Team

Novo Nordisk

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