Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1) (NCT07587021) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
36 participantsStarted 2026-06
Plain-language 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.
Who can participate
Age range
6 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)