Actively Recruiting

Early Phase 1
Age: 2Years +
All Genders
NCT06511349

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

128 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This study is a single-arm, open-label, single-dose, dose-escalation, and dose-expansion trial, aiming to assess the safety and tolerability of YOLT-203 in Chinese patients with Primary Hyperoxaluria Type 1 (PH1), and to preliminarily evaluate the effect of a single dose of YOLT-203 on plasma oxalate levels. In this study, the maximum duration of the screening period is 60 days, with the treatment day being Day 1 (D1), and the safety follow-up period extending to the 52nd week after dosing. Additionally, in the dose-escalation phase, after the first dose cohort, investigators will conduct a comprehensive evaluation based on safety, pharmacokinetic (PK), and pharmacodynamic (PD) data, and following discussion at the Safety Review Committee (SRC) meeting, subjects may voluntarily receive a second administration of the study drug at an effective dose level. After the completion of the main study, subjects will undergo long-term follow-up. In accordance with the requirements of the "Technical Guidance for Clinical Research on Long-term Follow-up of Gene Therapy Products (Trial)" issued by the Center for Drug Evaluation (CDE), long-term follow-up will be conducted for up to 15 years after dosing.

CONDITIONS

Official Title

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

Who Can Participate

Age: 2Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Participants aged 2 years or older at the time of signing informed consent
  • Diagnosed with primary hyperoxaluria type 1 (PH1) with AGXT gene mutations
  • Estimated glomerular filtration rate (eGFR) of at least 30 ml/min/1.73m2
  • At least two 24-hour urine collections showing oxalate excretion of 0.7 mmol/1.73m2/day or higher, or a single urine oxalate to creatinine ratio above the normal range for age
  • Stable vitamin B6 treatment for at least 90 days before enrollment, if receiving vitamin B6
  • Participant or guardian voluntarily signs informed consent
Not Eligible

You will not qualify if you...

  • Clinical evidence of systemic extra-renal oxalate deposition as judged by the investigator
  • Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels more than twice the upper limit of normal
  • Total bilirubin above 1.5 times the upper limit of normal, except for diagnosed Gilbert's syndrome with bilirubin less than twice the upper limit
  • International normalized ratio (INR) greater than 1.5, except for patients on oral anticoagulants with INR below 3.5
  • Active HIV infection or current/chronic hepatitis B or C virus infection
  • Estimated glomerular filtration rate (eGFR) below 30 ml/min/1.73m2
  • Use of investigational drugs within 30 days or 5 half-lives before study drug administration, or participation in another clinical study follow-up before randomization
  • History of kidney or liver transplantation
  • Other medical conditions or comorbidities that may interfere with study compliance or data interpretation
  • History of multiple drug allergies or allergic reactions to oligonucleotides or lipid nanoparticles (LNP)
  • History of intolerance to subcutaneous injections
  • Unwillingness to comply with contraceptive requirements during the study and for 6 months after
  • Female participants who are pregnant, planning pregnancy, or breastfeeding
  • Unwillingness or inability to limit alcohol consumption during the study; alcohol intake exceeding 2 units per day
  • History of alcohol abuse within 12 months before screening

AI-Screening

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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

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How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

1

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