ong Term Extension Study in Patients With Primary Hyperoxaluria
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 2 (PH2)Kidney DiseasesUrologic DiseasesGenetic DiseaseKidney DiseasesT51.2
- Registration Number
- LBCTR2020124677
- Lead Sponsor
- Dicerna Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Other
- Sex
- All
- Target Recruitment
- 50
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 60 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 formula by Schwartz in participants aged 6 to 16 years (Schwartz et al., 2009; National Kidney Foundation, 2002). In Japan, the formula by Uemura et al. will be used for participants aged 6 to 17 years (Uemura et al., 2014).
Key exclusion criteria include:
• Renal or hepatic transplantation; prior or planned within the study period
• 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 ame: To evaluate the effect of DCR PHXC on estimated glomerular filtration rate ;Timepoints: Annual change from baseline;Measure: estimated glomerular filtration rate
- Secondary Outcome Measures
Name Time Method ame: The incidence and severity of treatment-emergent adverse events (TEAE) and SAEs associated with abnormal 12 lead electrocardiogram (ECG) readings;Timepoints: TEAEs and SAEs are evaluated monthly for 3 years;Measure: Electrocardiogram (ECG)