YOLT-203 for Primary Hyperoxaluria Type 1: 6+ years old to adults

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 2 interventional trial evaluating the safety and efficacy of YOLT-203 in people with PH1, including participants aged 6 years and older. After an initial 6-month double-blind, placebo-controlled period, those on placebo will receive a single-dose YOLT-203 and vice versa for those on YOLT-203. The study uses randomized assignments to YOLT-203 or placebo, followed by a single-dose switch depending on initial group, to assess treatment effect while monitoring safety. Key eligibility includes confirmed PH1, stable vitamin B6 use, and specific urine oxalate criteria, with exclusions for significant extra-renal oxalate deposition and certain infections or pregnancy.

Why it matters

Who can join: the full rules

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).

Where it's running

• University of Alabama at Birmingham (UAB Hospital)
  Birmingham, Alabama, United States Recruiting
• Childrens Hospital of Colorado
  Aurora, Colorado, United States 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

Study contacts

• Study coordinator: Kim Marie Grabbe, MS, MBA
• Email: Kim.Marie.Grabbe@yoltx.com
• Phone: 217-851-5517

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Single-dose test of YOLT-203 in PH1 patients

  This study tests a single dose of YOLT-203 in people with Primary Hyperoxaluria Type 1 to see if it is safe and whether it lowers urinary oxalate.

  Recruiting Phase 1 YolTech Therapeutics Co., Ltd · Shanghai, Shanghai Municipality · NCT07776626

• Evaluating DCR-PHXC for patients with Primary Hyperoxaluria Type 1 and severe kidney disease

  This study is testing a new treatment called DCR-PHXC to see if it can help people with Primary Hyperoxaluria Type 1 and severe kidney disease feel better.

  Recruiting at 6 sites Phase 2 Novo Nordisk A/S · San Francisco, California and 17 other locations · NCT04580420

• National Registry for Rare Kidney Diseases

  This study is collecting health information from people with rare kidney diseases to help improve treatments and create better care guidelines.

  Recruiting UK Kidney Association · Bristol, South West · NCT06065852

• Biobank for patients with rare kidney stone diseases

  This study is collecting samples from patients with rare kidney stone diseases to help researchers learn more about these conditions and find better treatments to protect kidney health.

  Recruiting Mayo Clinic · Rochester, Minnesota · NCT02026388

• Registry for patients with rare kidney stone disorders

  This study is collecting health information from people with rare kidney stone disorders to better understand their symptoms and improve care for patients like them.

  Recruiting at 4 sites Mayo Clinic · Rochester, Minnesota and 3 other locations · NCT00588562

• Exploring YOLT-203 for treating Type 1 Primary Hyperoxaluria

  This study is testing a new treatment called YOLT-203 to see if it can safely lower oxalate levels in people with Type 1 Primary Hyperoxaluria.

  Recruiting Early Phase 1 RenJi Hospital · Shanghai, Shanghai Municipality · NCT06511349

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.