Study of YOLT-203 to Treat Type 1 Primary Hyperoxaluria in Patients Aged 2 and Older Evaluating Safety, Tolerability, and Effects on Plasma Oxalate Levels

What this Trial Is About

Researchers are evaluating the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 PH1. This early phase 1 trial also aims to preliminarily assess the effect of a single dose of YOLT-203 on plasma oxalate levels. The study includes a dose-escalation and dose-expansion design with detailed safety and pharmacokinetic assessments. Participants receive a single intravenous dose of YOLT-203 on Day 1 after a screening period of up to 60 days. In the dose-escalation phase, safety, pharmacokinetic, and pharmacodynamic data are reviewed before deciding if a second dose will be given at an effective dose level. After the main study, subjects enter a long-term follow-up period lasting up to 15 years to monitor ongoing safety and outcomes. Participants will be closely monitored with safety assessments through week 52, including blood tests to measure drug levels and effects over time. Pharmacodynamics and pharmacokinetics are evaluated up to day 28 and week 52 respectively. The study collects detailed data on tolerability, plasma oxalate changes, and long-term safety to understand the treatments impact and risks. The total participation includes screening, treatment, follow-up, and long-term monitoring phases.

Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)