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

Recruitment & Eligibility

Status: Recruiting

Sex: All

Target Recruitment: 7

Inclusion Criteria

Inclusion Criteria: - The age is = 2 years old at the time of signing the informed consent. - Have AGXT gene mutations and be diagnosed with primary hyperoxaluria (PH1); eGFR = 30 ml/min/1.73m2. - At least 2 times of 24-hour urinary oxalate excretion = 0.7 mmol/1.73m2/day or the ratio of urinary oxalate to creatinine in a single urine collection must be higher than the upper limit of normal (ULN) for the corresponding age. - If treated with vitamin B6, the treatment has been stable for 90 days before enrollment in the study and is willing to maintain the stable treatment plan unchanged during the study. - The patient himself/herself or the guardian voluntarily signs the informed consent. Exclusion Criteria: - The investigator judges that there is clinical evidence of systemic extra-renal oxalate deposition. - Have any of the following laboratory parameter assessment results at screening: 1. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 x the upper limit of normal (ULN). 2. Total bilirubin > 1.5 x ULN. If the increase in total bilirubin is caused by diagnosed Gilbert's syndrome and the total bilirubin < 2 x ULN, it is eligible. 3. International normalized ratio (INR) > 1.5 (Patients on oral anticoagulants [such as warfarin] and with INR < 3.5 will be allowed to participate). - Known to have active human immunodeficiency virus (HIV) infection; or have evidence of current or chronic hepatitis C virus (HCV) or hepatitis B virus (HBV) infection. - The estimated glomerular filtration rate (GFR) at screening is less than 30 mL/min/1.73m² (For patients = 18 years old, it will be calculated according to the Modification of Diet in Renal Disease [MDRD] formula; for patients < 18 years old, it will be calculated according to the Schwartz bedside formula). See the attachment. - Have received an investigational drug within the last 30 days or 5 half-lives (whichever is longer) before the first administration of the study drug, or have participated in the follow-up of another clinical study before randomization. - Have a history of kidney or liver transplantation. - According to the investigator's opinion, have other medical conditions or comorbidities that may interfere with study compliance or data interpretation. - Have a history of multiple drug allergies or allergic reaction history to oligonucleotides or LNP. - Have a history of subcutaneous injection intolerance. - Unwilling to comply with contraceptive requirements throughout the study participation period until 6 months after the end of the main study trial. - Female patients are pregnant, planning to become pregnant or breastfeeding. - Unwilling or unable to limit alcohol consumption throughout the study. Alcohol consumption during the study exceeds 2 units per day (1 unit: approximately 125 ml of wine = approximately 29 ml of spirits = approximately 284 ml of beer, will be excluded. - The investigator believes that there is a history of alcohol abuse within 12 months before screening.

Exclusion Criteria

Not provided

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability

Secondary Outcome Measures

Name	Time	Method
pharmacokinetics of YOLT-203;pharmacokinetics of YOLT-203;pharmacokinetics of YOLT-203;pharmacokinetics of YOLT-203;pharmacodynamics;pharmacodynamics;pharmacodynamics;pharmacodynamics

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