Contact SCGE




Gene Therapy Trial Report

Summary

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


NCTID NCT07587021 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication Primary Hyperoxaluria Type 1, PH1
Disease Ontology Term 🔄 DOID:0111670
Drug Product Name 🔄 YOLT-203
Sponsor YolTech Therapeutics Co., Ltd
Funder Type Industry
Recruitment Status
Not yet recruiting
Enrollment Count 36 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 HAO1
Therapeutic Modality 🔄 Gene editing
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Gene inactivation
Route of Administration 🔄 Intravenous
Drug Product Type 🔄 Encapsulated RNP
Gene Delivery System Type 🔄 Endocytosis
Non-Viral Vector Type LNP
Editor Type 🔄 YolCas12
Dose 1 🔄 Early Phase 1 doses: 0.3 mg/kg, 0.6 mg/kg, 1.0 mg/kg

Study Record Dates


Current Phase Phase2
Submit Date 2026-05-01
Completion Date 2028-02
Last Update 2026-05-14

Participation Criteria


Eligible Age >=6 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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).
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites
Locations

Regulatory Information


Has US IND True
FDA Designations 🔄 Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates US IND application cleared November 2025

Resources/Links