at San Francisco, California and other locations
study started
completion around



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.

Official Title

An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary Hyperoxaluria


Primary Hyperoxaluria Type 1 (PH1), Primary Hyperoxaluria Type 2 (PH2), Kidney Diseases, Urologic Diseases, Genetic Disease, Primary Hyperoxaluria Type 3 (PH3), Primary Hyperoxaluria, PH1, PH2, RNAi, GalNAc, LDHA, LDH, siRNA, PH3, Inborn Genetic Diseases, DCR-PHXC, Open Label


You can join if…

•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.

You CAN'T join if...

  • 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


  • Clinical Research Site
    San Francisco California 94143 United States
  • Clinical Trial Site
    Rochester Minnesota 55905 United States


accepting new patients by invitation only
Start Date
Completion Date
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Phase 3 research study
Study Type
Expecting 75 study participants
Last Updated