Primary hyperoxaluria
Conditions
Interventions
In adults and in adolescents (aged 12-17 years) weighing >=50 kg, DCR-PHXC will be administered once monthly at a dose of 170 mg. In adults and adolescents weighing < 50 kg, DCR-PHXC will be administe
Sponsors
Irina Yoffe
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR-PHXC. 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. 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 and two 24-hour Uox values >= 0.7 mmol (adjusted per 1.73 m2 body surface area [BSA]) at screening. OR for siblings aged 0 to 5 years old, average spot Uox-to-creatinine ratio at Screening above 2 times the 95th percentile for age based on Matos et al, 1999. - Estimated GFR at screening >= 30 mL/min normalized to 1.73 m2 body surface area (BSA). For infants aged less than 12 months, serum creatinine below the 97.5th percentile of a healthy population (Boer et al., 2010).
Exclusion criteria
Exclusion criteria: - Renal or hepatic transplantation; prior or planned within the study period - Plasma oxalate > 30 micromol/L - Current dialysis - Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The annual rate of decline in eGFR in participants with PH1 | — |
Countries
Australia, Canada, France, Germany, Italy, Japan, Lebanon, Netherlands, Spain, United Kingdom, United States
Contacts
Public Contactintellim inquiry desk for JRCT
intellim Corporation
Outcome results
None listed