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Gene-edited stem cells: a step toward transfusion-free life for β-thalassemia?

NCT ID NCT06479616

Disease control Sponsor: Children's Hospital of Fudan University Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This study follows 5 people with β-thalassemia who received CS-101, a gene-edited stem cell therapy, in an earlier trial. Researchers will monitor them for years to see if the treatment remains safe and allows them to stay free from blood transfusions. The goal is to understand whether this one-time therapy can provide lasting control of the disease.

What this could mean

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

Active substance
CS-101 (a gene-edited stem cell therapy)
What this could lead to
If successful, this could show that a single treatment with CS-101 allows people with β-thalassemia to live without needing regular blood transfusions.
What could go wrong
This is a very small follow-up study (5 people) with no control group, so results may not apply to everyone. Long-term risks like cancer or blood disorders are still being checked.

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.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

About 5 people

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

Started

Jun 2024

Expected to finish

Jun 2027

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.

Ages

3 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

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: * Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form * Participants must have received CS-101 infusion in last IIT study Exclusion Criteria: * There are no exclusion criteria

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Conditions

The condition(s) this trial relates to.

beta thalassemia

As listed by the trial registrant

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

Beta-thalassemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. 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 ↗

  3. 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.

Contacts and locations

Locations

  • Children's Hospital of Fudan University

    RECRUITING

    Shanghai, Shanghai Municipality, 201102, China

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