Find Primary Hyperoxaluria Clinical Trials Near You
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
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Genetic, Drug
Study Type: Interventional
Study Phase: Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• 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.
Locations
United States
Alabama
University of Alabama at Birmingham (UAB Hospital)
RECRUITING
Birmingham
Colorado
Childrens Hospital of Colorado
NOT_YET_RECRUITING
Aurora
Missouri
Washington University/Barnes Jewish Hospital
NOT_YET_RECRUITING
St Louis
Pennsylvania
University of Pennsylvania Health System (UPHS) - Penn Medicine
NOT_YET_RECRUITING
Philadelphia
Contact Information
Primary
Kim Marie Grabbe, MS, MBA
Kim.Marie.Grabbe@yoltx.com
217-851-5818
Backup
Claudia Rainolter
claudia.rainolter@yoltx.com
412-227-1722
Time Frame
Start Date:2026-09
Estimated Completion Date:2028-02
Participants
Target number of participants:36
Treatments
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.