Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
Primary Hyperoxaluria Type 1 (PH1)
About this trial
This is an expanded access trial for Primary Hyperoxaluria Type 1 (PH1)
Eligibility Criteria
Early Access Eligibility Criteria: Written, signed, and dated informed patient/parent consent; and for patients who were minors, age-appropriate assent (performed according to local regulations) Male or female patients at least 6 years of age Genetically-confirmed diagnosis of PH1 Accessible for treatment and follow-up and be able to comply with treatment monitoring requirements Not eligible for an ongoing RNAi therapy trial and not participating simultaneously in any interventional clinical research study Patients with PH1 that are not satisfactorily treated with current standard of care; i.e., 24-hour urinary oxalate (Uox) excretion ≥ 0.7 (millimoles) mmol for participants 18 years and older, or greater or lesser than (≥) 0.7 mmol per 1.73 metre square (m^2) body surface area (BSA) for participants less than 18 years of age Estimated GFR at screening ≥ 30 millilitres per minute (mL/min) normalized to 1.73 m^2 BSA No renal or hepatic transplantation; prior or planned within the treatment period No 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) Patient not currently on dialysis Plasma oxalate ≤ 30 micromoles per litre (μmol/L) Female patients not breastfeeding or pregnant The potential benefit for the individual patient justifies the potential risks of treatment as per prescribing physician judgment