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NCT07587021PHASE2Recruiting

Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)

YolTech Therapeutics Co., Ltd

Start Date

9/16/2026

Completion Date

2/1/2028

Summary

This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1. After the initial randomized, 6-month double-blind, placebo-controlled period, participants who were initially assigned to placebo will receive a single-dose of YOLT-203 treatment whereas participants in the YOLT-203 group will receive a single-dose of placebo infusion. For patients interested in additional information on how to participate, please follow this link: https://mytomorro.ws/ph1trial.

Eligibility Criteria

Age Range: 6 years to No maximum

Inclusion Criteria * You are 6 years old or older. * You have been diagnosed with PH1 (primary hyperoxaluria type 1) through genetic testing. * Urine tests show that your oxalate levels are not within certain limits (based on at least two 24-hour urine collections). * If you take vitamin B6, your dose has been stable for at least 3 months, and you are willing to keep it the same during the study. * You (and/or your parent or legal guardian, if you are under 18) understand the study and agree to follow all study requirements, including signing a consent form. Exclusion Criteria * You have signs that oxalate has affected parts of your body outside the kidneys. * Your liver blood tests are not within certain limits. * You have certain abnormal blood clotting results (unless controlled with medication like warfarin). * You have HIV, or active or chronic hepatitis C. * You are pregnant, planning to become pregnant, or breastfeeding. * You are not willing to use birth control during the study and for 6 months after (if applicable). * You are unable to limit alcohol intake to no more than about 2 drinks per day. * You have had alcohol or drug abuse in the past year. * You are unable to follow standard treatments for PH1 (such as drinking plenty of fluids, taking prescribed medications like potassium citrate, or following dietary advice).

Interventions

GENETIC

YOLT-203

DRUG

Placebo

Interested in This Trial?

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Conditions

Primary Hyperoxaluria Type 1PH1

Locations

University of Alabama at Birmingham (UAB Hospital)

Birmingham, Alabama 35249

United States

Childrens Hospital of Colorado

Aurora, Colorado 80045

United States

Washington University/Barnes Jewish Hospital

St Louis, Missouri 63108

United States

University of Pennsylvania Health System (UPHS) - Penn Medicine

Philadelphia, Pennsylvania 19107

United States