Phase I Exploratory Study of YOLT-203 in Patients With Primary Hyperoxaluria Type 1 (PH1)
This is a single-arm, open-label, single-dose dose-escalation study. It aims to evaluate the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily assess the effect of a single administration of YOLT-203 on 24-hour urinary oxalate excretion. The clinical trial will first evaluate the safety and tolerability in adult patients. After the safety profile is preliminarily confirmed, the trial will be sequentially conducted in adolescents, older children, and younger children.
• Subjects must meet ALL of the following criteria to be eligible for participation in this study:
• Age ≥ 6 years at the time of signing the informed consent form. Documented confirmation of Primary Hyperoxaluria Type 1 (PH1) diagnosis via genetic analysis prior to enrollment.
• Mean 24-hour urinary oxalate excretion level ≥ 0.7 mmol/24 h/1.73 m² obtained from at least two valid 24-hour urine collections.
• If the subject is receiving vitamin B6, the stable treatment regimen must have been maintained for a minimum of 90 days prior to enrollment, and the subject is willing to continue this regimen from administration of study drug through the end of the study.
• The subject is capable of understanding, willing, and able to comply with study requirements, and provides written informed consent. For subjects below the legal age of consent, a legal guardian must sign the informed consent form, and the subject shall provide assent in accordance with local and national requirements.