This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1. After the initial randomized, 6-month double-blind, placebo-controlled period, participants who were initially assigned to placebo will receive a single-dose of YOLT-203 treatment whereas participants in the YOLT-203 group will receive a single-dose of placebo infusion.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
36
Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)
Percent change from baseline in 24-hour urinary oxalate excretion corrected for body surface area (BSA) across Months 2 to 6.
Time frame: Months 2 to 6
Change From Baseline in Plasma Oxalate
Percent and absolute change from baseline in plasma oxalate levels across Months 2 to 6.
Time frame: Months 2 to 6
Change in Estimated Glomerular Filtration Rate (eGFR)
Change from baseline in estimated glomerular filtration rate (eGFR).
Time frame: Baseline to Month 6
Change in Kidney Stone Burden
Change from baseline in number and summed surface area of kidney stones as assessed by renal ultrasound.
Time frame: Baseline to Month 6
Proportion of Participants Achieving Urinary Oxalate Thresholds
Proportion of participants achieving urinary oxalate levels ≤ upper limit of normal (ULN) and ≤ 1.5 × ULN at Month 6.
Time frame: Month 6
Incidence of Adverse Events
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs), graded using CTCAE v6.0.
Time frame: Day 1 to Month 12
Pharmacokinetic Parameters of YOLT-203 (Cmax)
Cmax for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).
Time frame: Dosing through Month 1
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Pharmacokinetic Parameters of YOLT-203 (AUC0-inf)
AUC0-inf for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).
Time frame: Dosing through Month 1