[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100637819":3},{"organization":4,"outcomesModule":7,"designInfo":56,"detailedDescription":61,"studyPopulation":61,"armGroups":68,"interventions":81,"overallOfficials":92,"centralContacts":97,"locations":61,"responsibleParty":107,"collaborators":61,"id":109,"slug":61,"hasResults":110,"nctId":111,"briefTitle":112,"officialTitle":113,"acronym":61,"eligibilityCriteria":114,"healthyVolunteers":110,"sex":115,"minAge":116,"maxAge":61,"enrollmentInfo":117,"targetDuration":61,"studyType":120,"phases":121,"briefSummary":123,"conditions":124,"keywords":127,"overallStatus":141,"whyStopped":61,"lastUpdateSubmitDate":142,"lastUpdatePostDateStruct":143,"startDateStruct":146,"completionDateStruct":148,"leadSponsor":150,"locationsCount":61},{"fullName":5,"class":6},"YolTech Therapeutics Co., Ltd","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":39},[9],{"measure":10,"description":11,"timeFrame":12},"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",[14,17,21,24,28,32,36],{"measure":15,"description":16,"timeFrame":12},"Change From Baseline in Plasma Oxalate","Percent and absolute change from baseline in plasma oxalate levels across Months 2 to 6.",{"measure":18,"description":19,"timeFrame":20},"Change in Estimated Glomerular Filtration Rate (eGFR)","Change from baseline in estimated glomerular filtration rate (eGFR).","Baseline to Month 6",{"measure":22,"description":23,"timeFrame":20},"Change in Kidney Stone Burden","Change from baseline in number and summed surface area of kidney stones as assessed by renal ultrasound.",{"measure":25,"description":26,"timeFrame":27},"Proportion of Participants Achieving Urinary Oxalate Thresholds","Proportion of participants achieving urinary oxalate levels ≤ upper limit of normal (ULN) and ≤ 1.5 × ULN at Month 6.","Month 6",{"measure":29,"description":30,"timeFrame":31},"Incidence of Adverse Events","Incidence and severity of adverse events (AEs) and serious adverse events (SAEs), graded using CTCAE v6.0.","Day 1 to Month 12",{"measure":33,"description":34,"timeFrame":35},"Pharmacokinetic Parameters of YOLT-203 (Cmax)","Cmax for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).","Dosing through Month 1",{"measure":37,"description":38,"timeFrame":35},"Pharmacokinetic Parameters of YOLT-203 (AUC0-inf)","AUC0-inf for YOLT-203 components (mRNA, CRISPR RNA, and lipid nanoparticles).",[40,44,47,50,53],{"measure":41,"description":42,"timeFrame":43},"Health-Related Quality of Life","Change from baseline in EQ-5D Health State Profile and Visual Analog Scale (VAS) scores.","Baseline through Month 12",{"measure":45,"description":46,"timeFrame":20},"Change in urinary and plasma glycolate","Change from baseline in urinary and plasma glycolate levels.",{"measure":48,"description":49,"timeFrame":20},"Change in urinary oxalate:creatinine ratio","Change from baseline in urinary oxalate:creatinine ratio from spot urine samples.",{"measure":51,"description":52,"timeFrame":20},"Anti-Drug Antibody Incidence","Incidence of anti-drug antibodies (ADAs) against polyethylene glycol (PEG) and CRISPR-associated protein 12 (Cas12).",{"measure":54,"description":55,"timeFrame":20},"Kidney stone events","Number of kidney stone events occurring during the study period.",{"allocation":57,"interventionModel":58,"interventionModelDescription":59,"primaryPurpose":60,"observationalModel":61,"timePerspective":61,"maskingInfo":62},"RANDOMIZED","CROSSOVER","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.","TREATMENT",null,{"masking":63,"maskingDescription":61,"whoMasked":64},"TRIPLE",[65,66,67],"PARTICIPANT","CARE_PROVIDER","INVESTIGATOR",[69,75],{"label":70,"type":71,"description":72,"interventionNames":73},"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.",[74],"Genetic: YOLT-203",{"label":76,"type":77,"description":78,"interventionNames":79},"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.",[80],"Drug: Placebo",[82,88],{"type":83,"name":70,"description":84,"armGroupLabels":85,"otherNames":86},"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.",[70],[87],"Investigational Gene Editing Therapy",{"type":89,"name":76,"description":90,"armGroupLabels":91,"otherNames":61},"DRUG","Placebo is a matching intravenous infusion administered over 1 hour.",[76],[93],{"name":94,"affiliation":95,"role":96},"Ling Xiao, MD, PhD","YolTech Therapeutics","STUDY_DIRECTOR",[98,103],{"name":99,"role":100,"phone":101,"phoneExt":61,"email":102},"Kim Marie Grabbe, MS, MBA","CONTACT","217-851-5818","Kim.Marie.Grabbe@yoltx.com",{"name":104,"role":100,"phone":105,"phoneExt":61,"email":106},"Claudia Rainolter","412-227-1722","claudia.rainolter@yoltx.com",{"type":108,"investigatorFullName":61,"investigatorTitle":61,"investigatorAffiliation":61,"oldNameTitle":61,"oldOrganization":61},"SPONSOR","100637819",false,"NCT07587021","Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)","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","Inclusion Criteria\n\n* You are 6 years old or older.\n* You have been diagnosed with PH1 (primary hyperoxaluria type 1) through genetic testing.\n* Urine tests show that your oxalate levels are not within certain limits (based on at least two 24-hour urine collections).\n* 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.\n* You (and\u002For 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.\n\nExclusion Criteria\n\n* You have signs that oxalate has affected parts of your body outside the kidneys.\n* Your liver blood tests are not within certain limits.\n* You have certain abnormal blood clotting results (unless controlled with medication like warfarin).\n* You have HIV, or active or chronic hepatitis C.\n* You are pregnant, planning to become pregnant, or breastfeeding.\n* You are not willing to use birth control during the study and for 6 months after (if applicable).\n* You are unable to limit alcohol intake to no more than about 2 drinks per day.\n* You have had alcohol or drug abuse in the past year.\n* 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).","ALL","6 Years",{"count":118,"type":119},36,"ESTIMATED","INTERVENTIONAL",[122],"PHASE2","This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1.\n\nAfter 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.",[125,126],"Primary Hyperoxaluria Type 1","PH1",[128,129,130,131,132,133,134,70,135,136,137,138,139,140],"ph1","primary hyperoxaluria Type 1","yoltech","yolt203","primary hyperoxaluria","recurrent kidney stones","rare kidney stones","genetic kidney stones","gene editing","gene therapy","AGXT gene","CRISPR-Cas9","Alanine Glyoxylate Aminotransferase Gene","NOT_YET_RECRUITING","2026-05-07",{"date":144,"type":145},"2026-05-14","ACTUAL",{"date":147,"type":119},"2026-06",{"date":149,"type":119},"2028-02",{"name":5,"class":6}]