NCT07587021 · YolTech Therapeutics Co., Ltd
Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
What this study is about
This study will be conducted to evaluate the effectiveness and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.
View original scientific description
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.
Interventions
GENETIC
YOLT-203
YOLT-203 is an investigational gene editing therapy being evaluated for the treatment of Primary Hyperoxaluria Type 1 (PH1). It is administered by intravenous infusion over 1 hour.
DRUG
Placebo
Placebo is a matching intravenous infusion administered over 1 hour.
Primary outcome measures
Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)
Time frame: Months 2 to 6
Percent change from baseline in 24-hour urinary oxalate excretion corrected for body surface area (BSA) across Months 2 to 6.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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).
Where
- Birmingham, Alabama
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Aug 12, 2026 · Source of record for eligibility and locations