Recruiting
Phase 2

YOLT-203

Sponsor:

YolTech Therapeutics Co., Ltd

Code:

NCT07587021

Conditions

Primary Hyperoxaluria Type 1

PH1

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

Interventions

YOLT-203

Placebo

Study Details

Brief 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.

For patients interested in additional information on how to participate, please follow this link: https://mytomorro.ws/ph1trial.

Conditions

Primary Hyperoxaluria Type 1

PH1

Study ID

NCT07587021

Start date

Sep, 2026

Status verified date

Sep, 2026

Completion date

Feb, 2028

Anticipated

Primary completion date

Aug, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6+

Healthy Volunteers: Not accepted

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 Design

Enrollment

36 participants

Anticipated

Allocation

Randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: YOLT-203

Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive YOLT-203 during the double-blind treatment period and will cross over to the placebo treatment in the subsequent treatment period, followed by safety and efficacy follow-up.

placebo comparator: Placebo

Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive placebo during the double-blind treatment period and will cross over to the YOLT-203 treatment in the subsequent treatment period, followed by safety and efficacy follow-up.

Interventions

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.

Placebo

Placebo is a matching intravenous infusion administered over 1 hour.

Primary outcome measure

  • Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected) [ Time Frame: Months 2 to 6 ]

Central Contacts and Locations

Central contacts

Kim Marie Grabbe, MS, MBA

217-851-5818Kim.Marie.Grabbe@yoltx.com

Claudia Rainolter

412-227-1722claudia.rainolter@yoltx.com

Locations

University of Alabama at Birmingham (UAB Hospital)

Recruiting

Birmingham, Alabama, United States, 35249

Contacts

Crystal Taylor

205-934-3411crystalmtaylor@uabmc.edu

Principal Investigator:

Kyle Wood, MD

More Information

Sponsor

YolTech Therapeutics Co., Ltd

Last update posted

Sep 8, 2026

Last verified

Sep, 2026

Keywords

  • ph1
  • primary hyperoxaluria Type 1
  • yoltech
  • yolt203
  • primary hyperoxaluria
  • recurrent kidney stones
  • rare kidney stones
  • YOLT-203
  • genetic kidney stones
  • gene editing
  • gene therapy
  • AGXT gene
  • CRISPR-Cas9
  • Alanine Glyoxylate Aminotransferase Gene

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by YolTech Therapeutics Co., Ltd on 2026-09-08.