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

NCT07587021 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: PH1-YOLT-203-2001
• Study Type: interventional
• Target: 36
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Primary Hyperoxaluria Type 1; PH1

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

Outcome Measures

Primary Outcomes (1)

• Percent Change From Baseline in 24-Hour Urinary Oxalate (BSA-Corrected)

  Percent change from baseline in 24-hour urinary oxalate excretion corrected for body surface area (BSA) across Months 2 to 6.

  Months 2 to 6

Secondary Outcomes (7)

• Change From Baseline in Plasma Oxalate

  Months 2 to 6

• Change in Estimated Glomerular Filtration Rate (eGFR)

  Baseline to Month 6

• Change in Kidney Stone Burden

  Baseline to Month 6

• Proportion of Participants Achieving Urinary Oxalate Thresholds

  Month 6

• Incidence of Adverse Events

  Day 1 to Month 12

Other Outcomes (5)

• Health-Related Quality of Life

  Baseline through Month 12

• Change in urinary and plasma glycolate

  Baseline to Month 6

• Change in urinary oxalate:creatinine ratio

  Baseline to Month 6

Study Arms (2)

YOLT-203

EXPERIMENTAL

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.

Genetic: YOLT-203

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

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.

Also known as: Investigational Gene Editing Therapy

YOLT-203

Placebo DRUG

Placebo is a matching intravenous infusion administered over 1 hour.

Placebo

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• YolTech Therapeutics Co., Ltd: lead

MeSH Terms

Conditions

Primary hyperoxaluria type 1; Hyperoxaluria; Hyperoxaluria, Primary; Kidney Calculi

Condition Hierarchy (Ancestors)

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

Study Officials

• Ling Xiao, MD, PhD

  YolTech Therapeutics

  STUDY DIRECTOR

Central Study Contacts

Kim Marie Grabbe, MS, MBA

CONTACT

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

Claudia Rainolter

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: After the initial 6-month double-blind period, participants will enter a 6-month treatment extension 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.

Study Record Dates

• First Submitted: May 1, 2026
• First Posted: May 14, 2026
• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): February 1, 2028
• Last Updated: May 14, 2026

Record last verified: 2026-04