Ensayos clínicos

Key inclusion criteria include:

• 24-hour Uox excretion ≥  0.7 mmol (adjusted per 1.73 m2 body surface area [BSA] in participants < 18 years of age) in both collections performed in the screening period. Of the first 24 participants enrolled, at least 12 (50%) must have at least one 24‑hour Uox excretion ≥ 1.6 mmol (adjusted per 1.73 m2 BSA in participants aged < 18 years).
• Less than 20% variation between the two 24-hour urinary creatinine excretion values [mmol/24 hr/kg] derived from the two 24-hour urine collections in the screening period.
• Estimated glomerular filtration rate (eGFR) at screening ≥ 30 mL/min normalized to 1.73 m2 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 17 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 or anticipated requirement for dialysis during the study period
• Plasma oxalate > 30 µmol/L
• 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)
• Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >1.5 times upper limit of normal (ULN) for age and gender