[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"hutchinson-gilford-progeria-syndrome\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:hutchinson-gilford-progeria-syndrome":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,38],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":18,"targetDuration":21,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":28,"startDateStruct":31,"completionDateStruct":33,"leadSponsor":35,"locationsCount":19},"100657412","a-study-observing-disease-progression-in-a-patient-with-hutchinson-gilford-progeria-syndrome-hgps-100657412",false,"NCT07839351","A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)","A Prospective Longitudinal Observational Study of a Patient With Hutchinson-Gilford Progeria Syndrome: Establishing Natural History Data for the Development of AAV-Cas13-Based RNA-Targeting Gene Therapy","progeria","Inclusion Criteria:\n\n* A Hutchinson-Gilford Progeria Syndrome (HGPS) patient genetically confirmed by LMNA gene testing (c.1824C\\>T, p.G608G)\n\nExclusion Criteria:\n\n* not applicable","ALL",{"count":19,"type":20},1,"ESTIMATED","3 Years","OBSERVATIONAL","This study is a prospective, non-interventional, single-patient observational study following one patient with Hutchinson-Gilford Progeria Syndrome (HGPS), a rare disease that causes rapid, premature aging, over a 3-year period without any therapeutic intervention. The main purpose of this study is to track disease-related biomarkers over time - through physical measurements, cardiac and vascular ultrasound, bone age and bone density scans, cognitive testing, and quality-of-life assessments, along with blood tests (a total of 4 research blood draws, 3ml each) - in order to establish baseline natural history data that will support the future development and evaluation of an RNA-targeting gene therapy (based on AAV-Cas13 technology) for this condition.",[25],"Hutchinson-Gilford Progeria Syndrome","NOT_YET_RECRUITING","2026-09-21",{"date":29,"type":30},"2026-09-24","ACTUAL",{"date":32,"type":20},"2026-09",{"date":34,"type":20},"2032-12",{"name":36,"class":37},"Bundang CHA Hospital","OTHER",{"id":39,"slug":40,"hasResults":11,"nctId":41,"briefTitle":42,"officialTitle":43,"acronym":4,"eligibilityCriteria":44,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":45,"targetDuration":46,"studyType":22,"phases":4,"briefSummary":47,"conditions":48,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":49,"lastUpdatePostDateStruct":50,"startDateStruct":52,"completionDateStruct":54,"leadSponsor":56,"locationsCount":4},"100654697","creating-stem-cells-from-a-progeria-patients-blood-to-test-candidate-treatments-in-the-lab-100654697","NCT07803432","Creating Stem Cells From a Progeria Patient's Blood to Test Candidate Treatments in the Lab","A Case Study on the Establishment of Induced Pluripotent Stem Cell (iPSC) Lines From Hutchinson-Gilford Progeria Syndrome Patients and the In-Vitro Evaluation of Candidate Therapeutics","Inclusion Criteria\n\nClinically diagnosed with Hutchinson-Gilford Progeria Syndrome (HGPS) Currently followed as an outpatient at the study institution 19 years of age or younger at enrollment Able to provide written informed consent (or legal guardian able to provide consent on the patient's behalf) Willing and able to undergo a single peripheral blood draw for PBMC collection\n\nExclusion Criteria\n\nClinical condition precluding safe blood collection (e.g., severe anemia, active infection at the collection site, or a bleeding disorder) Patient or legal guardian unable or unwilling to provide informed consent Collected PBMCs yield insufficient quantity or viability for successful iPSC reprogramming (results in post-hoc exclusion from cell-based analysis only, not from study consent)",{"count":19,"type":20},"1 Year","This study aims to establish induced pluripotent stem cell (iPSC) lines from the blood of one pediatric patient with Hutchinson-Gilford Progeria Syndrome (HGPS), a rare disease that causes rapid, premature aging, in order to build a laboratory model of the disease. The main purpose of this study is to collect a blood sample (a total of 9cc, drawn once) from the patient, reprogram the patient's blood cells into stem cells, and then differentiate these stem cells into disease-relevant cell types (such as fibroblasts, vascular smooth muscle cells, and neurons) to study how HGPS affects cells at a molecular level. These patient-derived cells will then be used entirely in the laboratory (in-vitro) to test the safety and effectiveness of candidate treatments, including an RNA-targeting gene therapy, before any such therapy would be considered for use in patients.",[25],"2026-09-09",{"date":51,"type":30},"2026-09-10",{"date":53,"type":20},"2026-10",{"date":55,"type":20},"2029-12",{"name":36,"class":37}]