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Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
A Randomized, Double-blind, Placebo-controlled Study Followed by a Treatment Extension to Evaluate the Efficacy and Safety of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1
Who can join
Ages 6 and older · All sexes
Full eligibility criteria
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).
About the study
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.
What is being tested
- YOLT-203 (genetic)
- Placebo (drug)
Sponsor: YolTech Therapeutics Co., Ltd · Participants: 36 · Started: Sep 16, 2026
Contact the study team
- Kim Marie Grabbe, MS, MBA · Phone: 217-851-5818
- Claudia Rainolter · Phone: 412-227-1722
Official record on ClinicalTrials.gov — NCT07587021
Locations in the U.S.
| Alabama | University of Alabama at Birmingham (UAB Hospital), Birmingham |
| Colorado | Childrens Hospital of Colorado, Aurora (Not yet recruiting) |
| Missouri | Washington University/Barnes Jewish Hospital, St Louis (Not yet recruiting) |
| Pennsylvania | University of Pennsylvania Health System (UPHS) - Penn Medicine, Philadelphia (Not yet recruiting) |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.