[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"urea-cycle-disorders-inborn\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:urea-cycle-disorders-inborn":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,59,119],{"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":64,"acronym":4,"eligibilityCriteria":65,"healthyVolunteers":11,"sex":66,"minAge":67,"maxAge":68,"enrollmentInfo":69,"targetDuration":4,"studyType":24,"phases":71,"briefSummary":73,"conditions":74,"keywords":78,"overallStatus":108,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":116,"locationsCount":118},"100535742","phase-1-an-open-label-study-to-investigate-ecur-506-in-male-babies-less-than-9-months-of-age-with-neonatal-onset-otc-deficiency-100535742","NCT06255782","An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency","A Phase 1\u002F2\u002F3 First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less Than 9 Months of Age With Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency","Key Inclusion Criteria:\n\n1. Male sex\n2. Gestational or adjusted (corrected) gestational age ≥ 37 weeks\n3. Age at screening is 24 hours to 7 months\n4. Weight ≥ 3.5 kg and ≤ 13.5 kg at screening\n5. Has received age-appropriate vaccinations\n6. Genetically confirmed OTCD defined by genetic confirmation of an OTC variant (pathogenic or likely pathogenic) associated with severe neonatal OTCD defined below in Inclusion Criteria #7 or has the same OTC variant as a family member who had severe neonatal OTCD within first week of life.\n7. Severe neonatal OTCD defined by hyperammonemic crisis with elevated ammonia level of \\>560 μmol\u002FL and clinical symptoms within first week of life, and currently receiving treatment with both dietary protein restriction and nitrogen scavenger therapy.\n8. Current or historical biochemical profile consistent with OTCD\n9. Participant's parent(s)\u002FLAR must be able to comprehend and be willing to provide a signed IRB\u002FIEC-approved ICF.\n\nKey Exclusion Criteria:\n\n1. Neonatal diagnosis of severe to profound Hypoxic Ischemic Encephalopathy due to birth injury\n2. Requiring urgent liver transplant due to liver failure as assessed by the PI.\n3. Contiguous gene deletion involving the OTC gene and including at least the CYBB gene on the telomeric side or the TSPAN7 gene on the centromeric side.\n4. Known or suspected major organ injury\u002Fdysfunction\u002Fanomalies.\n5. Vital sign and laboratory abnormalities outside of reference ranges.\n6. Treatment with any other gene therapy or gene editing therapy\n7. Co-enrollment in any other study unless approved by the sponsor.\n8. Any condition, that in the opinion of the Investigator, would compromise the safety of the participant or study data\n9. Documented vertical transmission of HepA\u002FHepB\u002FHepC\n10. Documented in-utero teratogen, substance, and\u002For alcohol exposure, which in the opinion of the Investigator may increase the participant's risk of developmental delays, congenital anomalies, and\u002For significant medical complications","MALE","24 Hours","7 Months",{"count":70,"type":23},20,[72],"PHASE3","Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxifying of ammonia. Individuals with OTC deficiency can develop elevated levels of ammonia in the blood, potentially resulting in severe consequences, including cumulative and irreversible neurological damage, coma, and death. The most severe form presents shortly after birth and occurs more commonly in boys than girls.\n\nThis is a Phase 1\u002F2\u002F3, open-label, multicenter study evaluating the safety, efficacy, and dose of ECUR-506 in male babies with neonatal-onset OTC deficiency. The primary objective is to evaluate the safety, tolerability, and efficacy of up to three dose levels of ECUR-506 following intravenous (IV) administration of a single dose.",[75,76,77,30],"Ornithine Transcarbamylase Deficiency","Ornithine Transcarbamylase Deficiency Disease","Ornithine Carbamoyltransferase Deficiency (Disorder)",[79,80,81,82,83,84,85,86,87,88,89,90,91,92,93,94,95,96,97,98,99,100,101,75,102,103,104,105,106,31,107],"Amino Acid Metabolism, Inborn Errors","Ammonia","Brain Diseases","Brain Diseases, Metabolic","Brain Diseases, Metabolic, Inborn","Central Nervous System Diseases","Genetic Diseases, Inborn","Genetic Diseases, X-Linked","High Ammonia","Hyperammonemia","Inborn","Inborn Errors","Inherited Metabolic Disorders","Liver Disease","Liver Transplant","Metabolism","Metabolic Diseases","Metabolism, Inborn Errors","Neonatal","Nervous System Diseases","Neurometabolic disorders","NH4","Ornithine","OTC","OTC Deficiency","OTCD","Transcarbamylase","UCD","X-Linked","RECRUITING","2026-08-10",{"date":111,"type":50},"2026-08-12",{"date":113,"type":50},"2024-04-08",{"date":115,"type":23},"2027-12",{"name":117,"class":57},"iECURE, Inc.",12,{"id":120,"slug":121,"hasResults":11,"nctId":122,"briefTitle":123,"officialTitle":123,"acronym":4,"eligibilityCriteria":124,"healthyVolunteers":11,"sex":18,"minAge":68,"maxAge":125,"enrollmentInfo":126,"targetDuration":4,"studyType":128,"phases":4,"briefSummary":129,"conditions":130,"keywords":131,"overallStatus":108,"whyStopped":4,"lastUpdateSubmitDate":135,"lastUpdatePostDateStruct":136,"startDateStruct":138,"completionDateStruct":140,"leadSponsor":142,"locationsCount":58},"100578007","long-term-follow-up-ltfu-study-of-participants-in-any-iecure-protocol-using-an-investigational-product-ip-100578007","NCT06805695","Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)","Inclusion Criteria:\n\n1. Enrolled in an iECURE parent protocol and have either completed or discontinued that protocol\n2. Participant parent(s)\u002FLAR is willing and able to adhere to the protocol requirements.\n3. Consent was obtained by the participants parent(s)\u002FLAR (and participant assent, where applicable), prior to any study-related data being collected.\n\nExclusion Criteria:\n\n1\\. Participants who enroll into an interventional drug or gene therapy clinical trial utilizing an IP other than the IP provided in the parent protocol will be excluded from this protocol.","15 Months",{"count":127,"type":23},13,"OBSERVATIONAL","This LTFU is being conducted to assess long-term safety and durability of response in participants dosed with IP in a parent protocol, and to collect longitudinal natural history in enrolled but not dosed participants who also participated in a parent protocol.",[75,76,30],[79,80,81,82,83,84,85,86,87,88,89,90,92,93,94,95,96,97,98,100,101,75,102,103,104,105,106,31,132,133,134],"Long Term Follow Up","Long-Term Follow-Up","LTFU","2025-01-28",{"date":137,"type":50},"2025-02-03",{"date":139,"type":50},"2024-12-23",{"date":141,"type":23},"2041-07",{"name":117,"class":57}]