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Gene therapy aims to free patients from lifelong blood transfusions

NCT ID NCT06298630

Cure Sponsor: Bioray Laboratories 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 21, 2026 · Last updated Jul 22, 2026 · Updated 1 time

Summary

This study follows people who have already received an experimental gene therapy called BRL-101 for transfusion-dependent β-thalassemia, a severe blood disorder requiring regular transfusions. Researchers will monitor participants over the long term to check for safety issues, such as blood cancers, and to see if the treatment continues to work. The goal is to understand whether a single infusion of BRL-101 can provide lasting benefit.

What this could mean

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

Active substance
a gene therapy called BRL-101
What this could lead to
If successful, this could point toward a one-time gene therapy that frees people with transfusion-dependent β-thalassemia from needing regular blood transfusions.
What could go wrong
This is a small, early follow-up study, so the treatment may not prove safe or effective in the long run. There is also a risk of serious side effects, including 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.

Participants

About 45 people

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

Started

Dec 2024

Expected to finish

Oct 2038

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.

Who is studied

Subjects with transfusion-dependent β-thalassemia who have been treated with BRL-101.

Ages

3 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: 1. Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)/legal guardian(s) 2. Treated with BRL-101 for therapy of transfusion-dependent β-thalassemia. Exclusion Criteria: \- There are no exclusion criteria for this study

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia HBB/LCRB beta-thalassemia major

As listed by the trial registrant

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

Thalassemia, beta

How to take part

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

  1. The places running it

    6 sites. 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

  • Nanfang Hospital, Southern Medical University

    ACTIVE_NOT_RECRUITING

    Guangzhou, Guangdong, 510006, China

  • No. 923 Hospital of Joint Support Force of Chinese People 's Liberation Army Hospital

    NOT_YET_RECRUITING

    Nanning, Guangxi, 530021, China

  • Shenzhen Children 's Hospital

    NOT_YET_RECRUITING

    Shenzhen, Guangdong, 510006, China

  • Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

    NOT_YET_RECRUITING

    Guangzhou, Guangdong, 510006, China

  • The First Affiliated Hospital of GUANGXI MEDICAL UNIVERSITY

    RECRUITING

    Guangxi, 530021, China

  • Tianjin Institute of Hematology

    RECRUITING

    Tianjin, Tianjin Municipality, 300000, China

  • Xiangya Hospital of Central South University

    RECRUITING

    Changsha, Hunan, 510510, China

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