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

StatusNot Yet Recruiting
PhaseNot specified
Started
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Record History

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  1. Eligibility criteria shortened, about 23 words
  2. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT07803432
Lead Sponsor Bundang CHA Hospital
Collaborators: Korea Research Institute of Bioscience & Biotechnology
Conditions
(from sponsor)		 Hutchinson-Gilford Progeria Syndrome
Related terms Progeria
Enrollment 1 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov