Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Phase
Phase 3
Started
2019-07-09
Last updated
2025-05-30
Condition(s) studied
Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 2 (PH2)Kidney DiseasesUrologic DiseasesGenetic DiseasePrimary Hyperoxaluria Type 3 (PH3)
Investigational drug(s) / intervention(s)
DCR-PHXC
DCR-PHXC: Multiple fixed doses of DCR-PHXC by subcutaneous (SC) injection
Study summary
The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (\<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.
Eligibility
Sex
ALL
Min age
—
Max age
—
Healthy volunteers
No
Key Inclusion Criteria:
•Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC.
OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH.
* For participants rolling over from a multidose study of DCR-PHXC, enrollment should occur within a window of 25 to 75 days from the last dose of study intervention.
* Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula in participants aged ≥ 18 years, or the multivariate equation by Schwartz in participants aged 12 months to 17 years. In Japan, the cystatin C-based Uemura formula will be used for participants aged 12 months to \<2 years, the creatinine-based Uemura formula by will be used for participants aged 2 to 17 years, and the equation by Matsuo will be used in participants aged ≥ 18 years.
Key Exclusion Criteria:
* Renal or hepatic transplantation (prior or planned within the study period)
* Plasma oxalate \> 30 µmol/L
* Currently on dialysis
* Documented evidence of clinical manifestations of systemic oxalosis
Primary outcome measure(s)
The annual rate of decline in eGFR in participants with PH1 — Annual change from baseline To evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1
Trial sites (23)
Facility
City
Region
Status
Clinical Research Site
San Francisco
California
Clinical Trial Site
Boston
Massachusetts
Clinical Trial Site
Rochester
Minnesota
Clinical Trial Site
New York
New York
Clinical Research Site
Herston
Queensland
Clinical Trial Site
Melbourne
Australia
Clinical Research Site
Hamilton
Ontario
Clinical Trial Site
Bron
France
Clinical Trial Site
Paris
France
Clinical Trial Site
Bonn
Germany
Clinical Trial Site
Heidelberg
Germany
Clinical Research Site
Roma
Italy
Clinical Trial Site
Fukuoka
Japan
Clinical Trial Site
Nagoya
Japan
Clinical Trial Site
Tokyo
Japan
Clinical Trial Site
Beirut
Lebanon
Clinical Trial Site
Amsterdam
Netherlands
Clinical Trial Site
Tromsø
Norway
Clinical Research Site
Barcelona
Spain
Clinical Trial Site
Barcelona
Spain
Clinical Trial Site
Ankara
Turkey (Türkiye)
Clinical Trial Site
Hampstead
London
Clinical Trial Site
Birmingham
United Kingdom
More Dicerna Pharmaceuticals, Inc., a Novo Nordisk company trials in the UK
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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