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Can stem cells from a child with progeria reveal a path to therapy?

NCT ID NCT07803432

Knowledge-focused Sponsor: Bundang CHA Hospital Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 03, 2026 · Last updated Sep 04, 2026 · Updated 1 time

Summary

Researchers are taking a blood sample from one child with Hutchinson-Gilford progeria syndrome, a rare condition that causes rapid premature aging. They plan to turn those blood cells into stem cells and then into disease-relevant cells like muscle and nerve cells. This lab model will be used to test candidate treatments, including an RNA-targeting gene therapy, to see if they are safe and effective before any human use.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
RNA-targeting gene therapy (tested in the lab on patient-derived cells)
What this could lead to
If the lab model works, it could help screen treatments for progeria and point toward a therapy that slows or corrects the disease's effects.
What could go wrong
This is a single-patient lab study, not a treatment trial. The gene therapy is tested only on cells in a dish, so it may not work in the body or reach human trials.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 1 person

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Oct 2026

An estimate. Start dates often move.

Expected to finish

Dec 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Patients eligible for this study are those clinically diagnosed with Hutchinson-Gilford Progeria Syndrome (HGPS) who are currently followed as outpatients at the study institution, are 19 years of age or younger at enrollment, and are willing and able (or whose legal guardian is willing and able) to provide written informed consent and undergo a single peripheral blood draw for PBMC collection. Exclusion Criteria: * Patients are excluded if they have a clinical condition precluding safe blood collection (e.g., severe anemia, active infection at the collection site, or a bleeding disorder), if the patient or legal guardian is unable or unwilling to provide informed consent, or if the collected PBMCs yield insufficient quantity or viability for successful iPSC reprogramming (the latter resulting in post-hoc exclusion from cell-based analysis only, not from consent).

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Conditions

The condition(s) this trial relates to.

Hutchinson-Gilford progeria syndrome progeroid syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Hutchinson-Gilford progeria syndrome

How to take part

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  1. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  2. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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