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Gene therapy could free Beta-Thalassemia patients from lifelong transfusions

NCT ID NCT06308159

Disease control Sponsor: Lantu Biopharma 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tests a gene therapy for people with severe beta-thalassemia who need regular blood transfusions. The treatment modifies the patient's own blood stem cells to produce healthy hemoglobin, aiming to reduce or eliminate the need for transfusions. Six participants are being monitored for safety and effectiveness over time.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 6 people

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

Started

May 2024

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

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

Ages

Up 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: * Patients or parent(s)/legal guardian(s) willing and able to complete the informed consent process and comply with study procedures and visit schedules. * Diagnosis of beta-thalassemia and a history of RBCs transfusions. * Documented baseline, or pretransfusion, Hb≤7 g/dL. * Availability of an adequate and well-documented transfusion history. Exclusion Criteria: * Active bacterial, viral, fungal, or parasitic infection. * A white blood cell (WBC) counts\<3×10\^9/L, and/or platelet counts\<100×10\^9/L not related to hypersplenism. * Uncorrected bleeding disorder. * Presence of severe diseases that judged not compatible with the study procedures, such as severe hepatic disease, kidney disease, lung disease, and/or cardiovascular disease. * Uncontrolled seizure disorder. * Any evidence of severe iron overload that, in the investigator's opinion, warrants exclusion. * Prior autologous hematopoietic stem cell transplantation. * Prior receipt of gene therapy.

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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.

Beta-thalassemia

Contacts and locations

Locations

  • 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

    Kunming, Yunnan, 650100, China

  • Kunming Hope of Health Hospital

    Kunming, Yunnan, 650200, China

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