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Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
Sponsor: YolTech Therapeutics Co., Ltd
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.
Official 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
Key Details
Gender
All
Age Range
6 Years - Any
Study Type
INTERVENTIONAL
Enrollment
36
Start Date
2026-06
Completion Date
2028-02
Last Updated
2026-05-14
Healthy Volunteers
No
Conditions
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.