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Creating Stem Cells From a Progeria Patient's Blood to Test Candidate Treatments in the Lab

NCT07803432 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 1

Last updated 2026-09-03

No results posted yet for this study

Summary

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.

Conditions

  • Hutchinson-Gilford Progeria Syndrome

Sponsors & Collaborators

  • Korea Research Institute of Bioscience & Biotechnology

    collaborator OTHER_GOV

  • Bundang CHA Hospital

    lead OTHER

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-10-31
Primary Completion
2026-12-31
Completion
2029-12-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07803432 on ClinicalTrials.gov