This study is accepting new patients by invitation only

Long Term Extension Study in Patients With Primary Hyperoxaluria

a study on Hyperoxaluria Kidney Disease Urologic Diseases Genetic Diseases

Summary

Location
at San Francisco, California and other locations
Dates
study start
estimated completion

Description

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.

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

Keywords

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

Eligibility

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 (Levey & Stevens, 2010), or the multivariate equation by Schwartz in participants aged 12 months to 17 years (Schwartz et al., 2012). In Japan, the cystatin C-based formula by Uemura et al. will be used for participants aged 12 months to <2 years, the creatinine-based formula by Uemura et al. will be used for participants aged 2 to 17 years, and the equation by Matsuo et al. will be used in participants aged ≥ 18 years (Uemura et al., 2014a; Uemura et al., 2014b; Matsuo et al., 2009).

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

Locations

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

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
ID
NCT04042402
Phase
Phase 3 research study
Study Type
Interventional
Participants
Expecting 75 study participants
Last Updated