Actively Recruiting
Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)
Led by RenJi Hospital · Updated on 2025-05-16
21
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
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 treatment's impact and risks. The total participation includes screening, treatment, follow-up, and long-term monitoring phases.
CONDITIONS
Brief Title
Clinical Exploration Study of YOLT-203 in the Treatment of Type 1 Primary Hyperoxaluria (PH1)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 2 years or older at the time of informed consent
- Diagnosed with primary hyperoxaluria type 1 (PH1) with AGXT gene mutations
- Estimated glomerular filtration rate (eGFR) of 30 ml/min/1.73m2 or higher
- At least two 24-hour urinary oxalate tests showing elevated levels or a single urine oxalate-to-creatinine ratio above normal for age
- Stable vitamin B6 treatment for at least 90 days if applicable
- Voluntary informed consent signed by patient or guardian
You will not qualify if you...
- Clinical evidence of systemic extra-renal oxalate deposits
- Liver enzymes (ALT or AST) more than twice the normal upper limit
- Total bilirubin above 1.5 times the upper limit, except Gilbert's syndrome cases below 2 times
- International normalized ratio (INR) over 1.5 unless on anticoagulants with INR below 3.5
- Active HIV infection or current/chronic hepatitis B or C infection
- Estimated GFR below 30 ml/min/1.73m2
- Recent participation in another investigational drug study or follow-up within 30 days or 5 half-lives
- History of kidney or liver transplantation
- Other medical conditions impacting study compliance or data interpretation
- History of multiple drug allergies or allergic reactions to oligonucleotides or lipid nanoparticles
- History of subcutaneous injection intolerance
- Unwillingness to follow contraceptive requirements during and 6 months after study
- Pregnant, planning pregnancy, or breastfeeding
- Unable or unwilling to limit alcohol to 2 units per day during study
- History of alcohol abuse within 12 months before screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single dosing with follow-up through 28 days
Participants receive YOLT-203 intravenously at the predetermined dose for the treatment of type 1 primary hyperoxaluria (PH1).
1 dosing visit and multiple follow-up visits over 28 days
Duration - Up to 52 weeks
Participants are monitored for safety, tolerability, and pharmacodynamics through week 52 after treatment.
Regular follow-up visits through week 52
Trial Site Locations
Total: 1 location
1
Deparment of Liver Surgery, Ren Ji Hospital, School of Medicine, Shanghai Jiao Tong University
Shanghai, Shanghai Municipality, China, 200127
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
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