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

Status

Not yet recruiting

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07803432

Enrollment

Registered

2026-09-03

Start date

2026-10-01

Completion date

2029-12-01

Last updated

2026-09-10

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hutchinson-Gilford Progeria Syndrome

Brief 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.

Interventions

None listed

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Inclusion criteria

Clinically 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

Exclusion criteria

Clinical 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)

Design outcomes

Primary

Measure	Time frame	Description
Established an induced pluripotent stem cell (iPSC) cell line.	Through study completion, an estimated 12 months from PBMC collection	Established an induced pluripotent stem cell (iPSC) cell line using peripheral blood mononuclear cells (PBMCs) obtained from patients with Hutchinson-Gilford Progeria Syndrome.

Contacts

CONTACTMinYoung Kim

kmin@cha.ac.kr82-31-780-6281

Outcome results