Evaluation of the efficacy and safety of stiripentol in patients 6 years and older with primary hyperoxaluria type 1, 2 or 3

Trial status: Authorised Percent change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) from baseline to Month 3, Absolute change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) from baseline to Month 3 and Month 6, Change in 24-hour urine oxalate/creatinine ratio from baseline to Month 3 and Month 6, Proportion of patients with near normalisation of 24-hour urinary oxalate level corrected for BSA (defined as urinary oxalate lower than 1.5 x upper limit of normal (ULN)) at Month 3 and Month 6, Proportion of patients with normalisation of 24-hour urinary oxalate level corrected for BSA at Month 3 and Month 6, Change in estimated glomerular filtration rate (eGFR) from baseline to Month 6, Occurrence and frequency of kidney stone events during the follow-up, Change in urine oxalate/creatinine ratios as assessed in spot urine collections between baseline and Month 6, Change in biological parameters related to kidney stone formation between baseline and Month 6, Absolute change in the Pediatric Quality of Life Inventory (PedsQL [the generic and kidney failure (KF) modules]) for patients < 18 years of age (at screening) or in the Kidney Disease Quality of Life Questionnaire (KDQOL) for patients ≥ 18 years of age (at screening), Change in Euro Quality of Life Health State Profile Questionnaire (EQ-5D) and EQ-5D Visual Analog Scale (VAS) Percent change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) from baseline to Month 6

Percent change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) from baseline to Month 3, Absolute change in 24-hour urinary oxalate excretion corrected for body surface area (BSA) from baseline to Month 3 and Month 6, Change in 24-hour urine oxalate/creatinine ratio from baseline to Month 3 and Month 6, Proportion of patients with near normalisation of 24-hour urinary oxalate level corrected for BSA (defined as urinary oxalate lower than 1.5 x upper limit of normal (ULN)) at Month 3 and Month 6, Proportion of patients with normalisation of 24-hour urinary oxalate level corrected for BSA at Month 3 and Month 6, Change in estimated glomerular filtration rate (eGFR) from baseline to Month 6, Occurrence and frequency of kidney stone events during the follow-up, Change in urine oxalate/creatinine ratios as assessed in spot urine collections between baseline and Month 6, Change in biological parameters related to kidney stone formation between baseline and Month 6, Absolute change in the Pediatric Quality of Life Inventory (PedsQL [the generic and kidney failure (KF) modules]) for patients < 18 years of age (at screening) or in the Kidney Disease Quality of Life Questionnaire (KDQOL) for patients ≥ 18 years of age (at screening), Change in Euro Quality of Life Health State Profile Questionnaire (EQ-5D) and EQ-5D Visual Analog Scale (VAS)

Age group: 65+ years, 18-64 years, 0-17 years Gender: Female, Male Target enrollment: 19 participants