Hutchinson-Gilford Progeria SyndromeNCT07803432
Creating Stem Cells From a Progeria Patient's Blood to Test Candidate Treatments in the Lab
An observational study of Hutchinson-Gilford Progeria Syndrome, run by Bundang CHA Hospital.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hutchinson-Gilford Progeria Syndrome
- How many people
- 1 participants
- Where
- No sites listed yet
- Study dates
- Oct 2026to Dec 2029
- How long will I take part?
- 1 Year
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Study population: This study will enroll a single pediatric patient with Hutchinson-Gilford Progeria Syndrome (HGPS) who is currently receiving outpatient follow-up care at the Department of Rehabilitation Medicine, CHA Bundang Medical Center. The patient was identified through routine clinical care at this institution; no external recruitment or advertisement will be used.
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: 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
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
- Medical subject headings
- Progeria
What is being measured
The primary outcome is the main question the study is designed to answer.
Established an induced pluripotent stem cell (iPSC) cell line.
Established an induced pluripotent stem cell (iPSC) cell line using peripheral blood mononuclear cells (PBMCs) obtained from patients with Hutchinson-Gilford Progeria Syndrome.
Measured: Through study completion, an estimated 12 months from PBMC collection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Sep 2026
- Study startOct 2026 (estimated)
- Primary completionDec 2026 (estimated)
- Study completionDec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hutchinson-Gilford Progeria SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 1 participants (estimated)
- Target duration
- 1 Year
Sponsor and investigators
- Lead sponsor
- Bundang CHA Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Korea Research Institute of Bioscience & Biotechnology
- Responsible party
- Principal investigator, MinYoung Kim, MD, PhD, Bundang CHA Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07803432
- Sponsor's protocol ID
- 2025-10-012
- Status last verified
- Sep 2026
- First submitted
- 31 Aug 2026
- Last updated
- 10 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07803432
- Acronym / sponsor ID
- 2025-10-012
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 1 participants (estimated)
- Lead sponsor
- Bundang CHA HospitalUniversity, hospital or other
- Collaborators
- Korea Research Institute of Bioscience & Biotechnology
- Study start
- Oct 2026 (estimated)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Dec 2029 (estimated)
- First posted
- 3 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Target follow-up
- 1 Year
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- This study will enroll a single pediatric patient with Hutchinson-Gilford Progeria Syndrome (HGPS) who is currently receiving outpatient follow-up care at the Department of Rehabilitation Medicine, CHA Bundang Medical Center. The patient was identified through routine clinical care at this institution; no external recruitment or advertisement will be used.
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (1 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Established an induced pluripotent stem cell (iPSC) cell line.
Time frame: 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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Sep 2026.
Related trials for Hutchinson-Gilford Progeria Syndrome
Not yet recruitingObservational study
A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)
Hutchinson-Gilford Progeria Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
An observational study of Hutchinson-Gilford Progeria Syndrome, run by Bundang CHA Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 10 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact MinYoung Kim (82-31-780-6281, kmin@cha.ac.kr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Established an induced pluripotent stem cell (iPSC) cell line. (time frame: Through study completion, an estimated 12 months from PBMC collection). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Sep 2026.