[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"nags-deficiency\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:nags-deficiency":34},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,59],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":17,"sex":18,"minAge":19,"maxAge":20,"enrollmentInfo":21,"targetDuration":4,"studyType":24,"phases":25,"briefSummary":28,"conditions":29,"keywords":41,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":51,"completionDateStruct":53,"leadSponsor":55,"locationsCount":58},"100652398","phase-1-phase-12-study-of-krro-121-in-healthy-volunteers-and-patients-with-ucd-100652398",false,"NCT07773246","Phase 1\u002F2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD","A Phase 1\u002F2, Randomized, Double-Blind, Placebo-Controlled, Single- and Multiple-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of KRRO-121 in Healthy Volunteers and Patients With Urea Cycle Disorders","ANCHOR","Inclusion Criteria:\n\n* Adult male or female participants, 18 to 65 years of age\n* Healthy as determined by medical history, physical examination, vital signs, 12-lead electrocardiogram (ECG), and screening laboratory assessments, in the opinion of the Investigator.\n* Female participant of childbearing potential or male participant that is willing to use an approved, reliable means of contraception\n* Willing and able to provide signed written informed consent and to comply with all study requirements\n\nExclusion Criteria:\n\n* Any medical history that is clinically significant in the opinion of the Principal Investigator and Sponsor Medical Monitor\n* Any laboratory value at screening or predose on Day -1 outside the local laboratory reference range that is clinically significant in the opinion of the Principal Investigator and Sponsor Medical Monitor\n* Evidence of active systemic infection at Screening or Day 1 requiring antimicrobial, antiviral, or antifungal therapy\n* Use of any investigational drug or device within 3 months or 5 half-lives (whichever is longer) prior to Day 1\n* Concurrent participation in any other interventional clinical study",true,"ALL","18 Years","65 Years",{"count":22,"type":23},30,"ESTIMATED","INTERVENTIONAL",[26,27],"PHASE1","PHASE2","Phase 1\u002F2 Study of KRRO-121 is a randomized, placebo-controlled study in Healthy Volunteers (Phase 1 SAD and MAD) and Study Participants with Urea Cycle Disorders (Phase 2). The primary objective of the study is to evaluate the safety of KRRO-121.",[30,31,32,33,34,35,36,37,38,39,40],"Urea Cycle Disorders, Inborn","Urea Cycle Disorders","Ornithine Transcarbamylase (OTC) Deficiency","Arginase 1 Deficiency","NAGS Deficiency","Carbamylphosphate Synthetase I","Ornithine Transporter Mitochondrial 1","Argininosuccinate Synthase","Argininosuccinate Lyase Deficiency","N-acetyl Glutamate - Synthetase","Citrin Deficiency",[42,43,44,45],"KRRO-121","stabilized variant of glutamine synthetase (GS)","ammonia clearance","RNA-editing","NOT_YET_RECRUITING","2026-08-18",{"date":49,"type":50},"2026-08-20","ACTUAL",{"date":52,"type":23},"2026-09",{"date":54,"type":23},"2027-09",{"name":56,"class":57},"Korro Bio, Inc.","INDUSTRY",1,{"id":60,"slug":61,"hasResults":11,"nctId":62,"briefTitle":63,"officialTitle":63,"acronym":4,"eligibilityCriteria":64,"healthyVolunteers":11,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":65,"targetDuration":4,"studyType":67,"phases":4,"briefSummary":68,"conditions":69,"keywords":79,"overallStatus":84,"whyStopped":4,"lastUpdateSubmitDate":85,"lastUpdatePostDateStruct":86,"startDateStruct":88,"completionDateStruct":90,"leadSponsor":92,"locationsCount":5},"100432223","hepatic-histopathology-in-urea-cycle-disorders-100432223","NCT04908319","Hepatic Histopathology in Urea Cycle Disorders","Inclusion Criteria:\n\n* Diagnosis of primary urea cycle disorder based on clinical suspicion confirmed by enzyme activity, DNA testing or metabolite analysis.\n* History of liver transplantation and\u002For liver biopsy OR\n* Planned liver transplantation and\u002For liver biopsy\n\nExclusion Criteria:\n\n* Unavailability of histopathology report from the liver biopsy or explant, or unavailability of liver tissue or slides from the biopsy or explant OR\n* Anticipated inability to obtain pathology report, liver disease, tissue blocks, or pathology slides after liver biopsy or transplantation\n* Known history of a secondary cause for liver disease such as chronic viral hepatitis, autoimmune liver disease, short gut, small bowel syndrome, alcohol liver disease, or TPN-related cholestatic disease",{"count":66,"type":23},70,"OBSERVATIONAL","This is a multi-site, retrospective chart review as well as a prospective study to evaluate histopathologic findings in liver samples from individuals with any UCD diagnosis. This study will be conducted at all Urea Cycle Disorders Consortium (UCDC) sites: Baylor College of Medicine in Houston, TX and Children's National Medical Center in Washington D.C.",[70,71,72,73,74,75,76,77,78,34],"Urea Cycle Disorder","Ornithine Transcarbamylase Deficiency","Citrullinemia 1","ARGI Deficiency","ASL Deficiency","Argininosuccinic Aciduria","ASS Deficiency","Hyperargininemia","Carbamyl Phosphate Synthetase Deficiency",[80,81,82,83],"Liver transplant","Liver disease","Liver biopsy","Histopathology","RECRUITING","2025-07-17",{"date":87,"type":50},"2025-07-20",{"date":89,"type":50},"2022-02-24",{"date":91,"type":23},"2026-06-30",{"name":93,"class":94},"Baylor College of Medicine","OTHER"]