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NCT07587021From ClinicalTrials.govRecruiting

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

  • Primary Hyperoxaluria Type 1
  • PH1

At a glance

Phase
Phase 2
Study type
Interventional
Sponsor
YolTech Therapeutics Co., Ltd
Enrolment target
36
Started
16 September 2026
Main results due
August 2027
Study sites
4
Registry updated
23 September 2026

Can you take part?

  • Aged 6 years and over.
  • Open to any sex.
  • You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the 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).

What this study is about

In the sponsor’s own words, from the registry.

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.

Study sites(4)

  • University of Alabama at Birmingham (UAB Hospital)

    Birmingham, Alabama, United States

    Recruiting

  • Childrens Hospital of Colorado

    Aurora, Colorado, United States

    Not yet recruiting

  • Washington University/Barnes Jewish Hospital

    St Louis, Missouri, United States

    Not yet recruiting

  • University of Pennsylvania Health System (UPHS) - Penn Medicine

    Philadelphia, Pennsylvania, United States

    Not yet recruiting

Showing 4 of 4 sites. 1 of the 4 sites on this study is recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT07587021. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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