ong Term Extension Study in Patients With Primary Hyperoxaluria (PHYOX3)
- Conditions
- Primary hyperoxaluria
- Registration Number
- JPRN-jRCT2041200091
- Lead Sponsor
- Verity Rawson
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 5
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).
- 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)
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method