ClinicalTrials.gov record NCT07587021

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

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 2A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
Age6 years old and older
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This randomized, triple-blind study tests a single intravenous dose of YOLT-203 in children and people with primary hyperoxaluria type 1 (PH1). The first 6 months are a double-blind, placebo-controlled period; afterward participants cross over so both groups receive the alternate infusion. The study collects measures of urinary and plasma oxalate, kidney function, stone burden, drug levels, and safety.

What it is studying. The study is evaluating whether a single intravenous infusion of YOLT-203 changes 24-hour urinary oxalate excretion (body-surface-area corrected) compared with placebo over Months 2–6, and is measuring safety, plasma oxalate, kidney function (eGFR), kidney stone burden, adverse events, and pharmacokinetics.

What the researchers hope to learn. To learn whether YOLT-203 affects urinary and plasma oxalate levels and to assess its safety, effects on kidney function and stone burden, and pharmacokinetic properties in people with PH1.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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 being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • Placebo
Genetic
  • YOLT-203

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

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).

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomized in a triple‑masked, 6‑month double‑blind period to receive a single intravenous (IV) 1‑hour infusion of YOLT‑203 or matching placebo; after that period participants cross over so those first on placebo receive YOLT‑203 and those first on YOLT‑203 receive placebo. Study visits include repeated 24‑hour urine collections, blood tests including plasma oxalate (measured Months 2-6) and pharmacokinetics (through Month 1), kidney ultrasound to assess stone burden, and safety monitoring with adverse‑event reporting (through Month 12). You will be asked to keep vitamin B6 dose stable if applicable, follow standard PH1 care instructions, limit alcohol as directed, and follow contraception requirements if applicable.

How the study is structured. Participants are randomly assigned and the study is triple-blind (participants, care teams, and investigators do not know assignments). The initial period is a 6-month double-blind, placebo-controlled phase. After that, participants cross over so those first given placebo receive a single dose of YOLT-203 and those first given YOLT-203 receive a single-dose placebo in a subsequent period. YOLT-203 and placebo are given as a one-hour intravenous infusion; safety and outcomes are followed through about Month 12.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on primary hyperoxaluria

Clinical trials13 trials listed for primary hyperoxaluriaEvery trial the registry lists for primary hyperoxaluria, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in primary hyperoxaluria researchLatest monthly update: September 2026. Includes: Early oxalate change. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Sep 16, 2026
Primary completion
August 2027 (estimated)
Study completion
February 2028 (estimated)
Target enrollment
36 participants (estimated)
Last updated on registry
Oct 9, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
YolTech Therapeutics Co., Ltd

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (4)

Terms used on this page · explanations by MedicalUpdates.ai

randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
legal guardian
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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