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Can a single stem cell infusion rewrite the code of beta thalassemia?

NCT ID NCT03351829

Cure Sponsor: Shenzhen Geno-Immune Medical Institute Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 27, 2026 · Last updated Aug 28, 2026 · Updated 1 time

Summary

This trial tests whether a person's own blood stem cells, modified in the lab to carry a corrected gene, can cure beta thalassemia. Participants receive one or more infusions of these gene-modified cells after a mild conditioning regimen. The goal is to see if the cells safely produce healthy red blood cells and reduce or eliminate the need for transfusions.

What this could mean

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

Active substance
Gene-modified autologous hematopoietic stem cells
What this could lead to
If successful, this could offer a one-time treatment that frees people with beta thalassemia from lifelong blood transfusions and iron-chelation therapy.
What could go wrong
This is an early-stage trial with only 20 participants, so safety and effectiveness are still unproven. Risks include immune reactions, graft failure, and a small chance of gene insertion causing blood cancers.

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 20 people

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

Expected to start

Dec 2026

An estimate. Start dates often move.

Expected to finish

Dec 2030

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

4 to 70 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: 1. Diagnosis of Beta Thalathemia. 2. Age: ≥ 4 years. 3. Karnofsky: ≥ 80%. 4. Left ventricular ejection fraction (LVEF): \> 50%; no obvious heart disease and pulmonary hypertension. 5. Pulmonary function is normal; forced expiratory volumein one second (FEV1) and vital capacity greater than 60% and DLCO \> 50%. 6. Serum creatinine ≤ 2 × upper limit of normal range. 7. MRI showed no super-iron load in the heart and liver, and no severe cirrhosis. 8. Normal Coagulation. 9. Written, informed consent obtained prior to any study-specific procedures. Exclusion Criteria: 1. Diagnosis of active malignant disease (other than Bowen disease or cervical cancer); or has family history of cancer. 2. Myelopathy, tumor-related cytogenetic changes or other more severe blood diseases. 3. Has alcoholism experience within 6 months prior to enrollment. 4. History of epilepsy. 5. History of bone marrow transplantation. 6. Existence of an available HLA-identical related donor. 7. Pregnant or lactating females. 8. Subject infected with HIV (HIV antibody positive), Treponema pallidum antibody positive or TB culture positive. 9. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.

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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

  • Shenzhen Geno-immune Medical Institute

    Shenzhen, Guangdong, 518000, China

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