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New stem cell therapy aims to free patients from lifelong blood transfusions

NCT ID NCT04390971

Disease control Sponsor: Institute of Hematology & Blood Diseases Hospital, China 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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a new treatment called ET-01 in 3 people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular blood transfusions. The treatment uses the patient's own stem cells, which are modified and then transplanted back. The goal was to see if it is safe and can reduce or eliminate the need for transfusions by helping the body produce healthy red blood cells.

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

3 people

The number who actually took part.

Started

Feb 2023

Finished

Mar 2023

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

6 to 35 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: * Subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms; * 6\~35 years old, all gender; * Diagnosis of transfusion dependent β-thalassemia (β-TDT) as defined by protocol; * Eligible for autologous stem cell transplant; * Eligible for autologous stem cell transplant; * Organs in good function. * Other protocol defined inclusion criteria may apply. Exclusion Criteria: * Subjects with associated α-thalassemia; * Active bacterial, virus, fungal or parasitic infection, including HIV infection, HbsAg and HBV DNA positive, HCV DNA positive, or Treponema Pallidum infection; * HLA identical sibling or unrelated donors are available; * Prior allo-HSCT or gene therapy. * Other protocol defined exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major

As listed by the trial registrant

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

Transfusion dependent beta-thalassaemia

Contacts and locations

Locations

  • Affiliated Hospital of Zunyi Medical University

    Zunyi, Guizhou, 563000, China

  • Institute of Hematology & Blood Diseases Hospital

    Tianjin, Tianjin Municipality, 300020, China

  • The Affiliated Hospital of Guizhou Medical University

    Guiyang, Guizhou, 550004, China

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