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NCT Number: NCT07587021

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

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.

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Key information

Conditions

Primary Hyperoxaluria Type 1 Calculi Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hyperoxaluria Hyperoxaluria, Primary Kidney Calculi Kidney Diseases Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nephrolithiasis Nutritional and Metabolic Diseases PH1 Pathological Conditions, Anatomical Pathological Conditions, Signs and Symptoms Urinary Calculi Urogenital Diseases Urolithiasis Urologic Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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).

Treatment and study plan

YOLT-203

Genetic

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.

Other names: Investigational Gene Editing Therapy

Placebo

Drug

Placebo is a matching intravenous infusion administered over 1 hour.

Primary outcomes

  1. 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.

Secondary outcomes

  1. Change From Baseline in Plasma Oxalate

    Time frame: Months 2 to 6

    Percent and absolute change from baseline in plasma oxalate levels across Months 2 to 6.

  2. Change in Estimated Glomerular Filtration Rate (eGFR)

    Time frame: Baseline to Month 6

    Change from baseline in estimated glomerular filtration rate (eGFR).

  3. Change in Kidney Stone Burden

    Time frame: Baseline to Month 6

    Change from baseline in number and summed surface area of kidney stones as assessed by renal ultrasound.

  4. Proportion of Participants Achieving Urinary Oxalate Thresholds

    Time frame: Month 6

    Proportion of participants achieving urinary oxalate levels ≤ upper limit of normal (ULN) and ≤ 1.5 × ULN at Month 6.

  5. Incidence of Adverse Events

    Time frame: Day 1 to Month 12

    Incidence and severity of adverse events (AEs) and serious adverse events (SAEs), graded using CTCAE v6.0.

  6. Pharmacokinetic Parameters of YOLT-203 (Cmax)

    Time frame: Dosing through Month 1

    Cmax for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).

  7. Pharmacokinetic Parameters of YOLT-203 (AUC0-inf)

    Time frame: Dosing through Month 1

    AUC0-inf for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).

Other outcomes

  1. Health-Related Quality of Life

    Time frame: Baseline through Month 12

    Change from baseline in EQ-5D Health State Profile and Visual Analog Scale (VAS) scores.

  2. Change in urinary and plasma glycolate

    Time frame: Baseline to Month 6

    Change from baseline in urinary and plasma glycolate levels.

  3. Change in urinary oxalate:creatinine ratio

    Time frame: Baseline to Month 6

    Change from baseline in urinary oxalate:creatinine ratio from spot urine samples.

  4. Anti-Drug Antibody Incidence

    Time frame: Baseline to Month 6

    Incidence of anti-drug antibodies (ADAs) against polyethylene glycol (PEG) and CRISPR-associated protein 12 (Cas12).

  5. Kidney stone events

    Time frame: Baseline to Month 6

    Number of kidney stone events occurring during the study period.

Study contacts

Contact information is provided by the study sponsor or research team.

Claudia Rainolter

CONTACT

[email protected]

412-227-1722

Kim Marie Grabbe, MS, MBA

CONTACT

[email protected]

217-851-5818

Sponsors and collaborators

Lead sponsor

YolTech Therapeutics Co., Ltd

Industry

Registry information

Official study title

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

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
May 14, 2026
Registry last updated
May 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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