Status:
ACTIVE_NOT_RECRUITING
Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)
Lead Sponsor:
Guangzhou Women and Children's Medical Center
Conditions:
Type 1 Primary Hyperoxaluria
Eligibility:
All Genders
2-18 years
Phase:
EARLY_PHASE1
Brief Summary
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)...
Eligibility Criteria
Inclusion
- The age is 2≤ years \<18 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
- The investigator judges that there is clinical evidence of systemic extrarenal oxalate deposition.
- Have any of the following laboratory parameter assessment results at screening:
- Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \> 2 x the upper limit of normal (ULN).
- 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.
- 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 halflives (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.
- Page 4 of 5 \[DRAFT\] -
- 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.
Key Trial Info
Start Date :
February 27 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 1 2026
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT06892301
Start Date
February 27 2025
End Date
July 1 2026
Last Update
March 24 2025
Active Locations (1)
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1
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, China, 510623