This study is a single-arm, open-label, single-dose, dose-escalation trial, aiming to evaluate the safety and tolerability of YOLT-203 in the Chinese population with type 1 primary hyperoxaluria (PH1); and to preliminarily assess the effect of a single dose of YOLT-203 on the plasma oxalate level.In this study, the maximum screening period of the main study is 60 days, the treatment day is Day 1 (D1), and the safety follow-up period is up to Week 52 after administration. In addition, subjects within the first dose group can voluntarily receive a second treatment with the test drug at the effective dose level. After the end of the main study, the subjects will undergo long-term followup. According to the requirements of the "Technical Guidelines for Long-Term Follow-up Clinical Studies of Gene Therapy Products (Trial)" issued by the CDE, the long-term follow-up is up to 15 years after administration. The most updated protocol is V1.2 , 22 Jan 2025
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
2
The IP is administered intravenously at the predetermined dose.
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, China
Incidence of Treatment-Emergent Adverse events
An Adverse Event(AE) is any untoward medical occurrence in a participant or clinical investigational participant adminstered a medicinal product and which does not necessarily have a casual relationship with this treatment
Time frame: through week 52
The Peak Plasma Concentration (Cmax) of YOLT-203
The Peak Plasma Concentration (Cmax) of YOLT-203 in the plasma after a dose is given
Time frame: through Day 14
Area under the plasma concentration versus time curve (AUC)of YOLT-203
Area under the plasma concentration versus time curve (AUC). 0.5 predose,6,24,48,144,and312 hours after treatment
Time frame: through Day 14
Time to Maximum Plasma Concentration (Tmax)of YOLT-203
Tmax is the time it takes for YOLT-203 to reach the maximun concentration (Tmax) after administration
Time frame: through Day 14
Drug half-life (T1/2)of YOLT-203
The amount of time it takes for the plasma YOLT-203 drug concentration to drop to half
Time frame: through Day 14
The changes in blood glycolic acid levels
After medication, at 1, 2, 4, 8, 16, 24, 36, and 52 weeks, the changes in blood glycolic acid levels
Time frame: through week 52
The changes in 24-hour urinary oxalic acid excretion
After medication, at 1,2,4, 8, 16, 24, 36and 52 weeks, the changes in 24-hour urinary oxalic acid excretion compared to the baseline value.
Time frame: through week 52
The changes in 24-hour urinary glycolic acid excretion
After medication, at 1, 2, 4, 8, 16, 24, 36, and 52 weeks, the changes in 24-hour urinary glycolic acid excretion
Time frame: through week 52
The changes in eGFR
After medication, at 1, 2, 4, 8, 16, 24, 36, and 52 weeks, the changes in eGFR.
Time frame: through week 52
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