Gene therapy aims to free patients from lifelong blood transfusions
NCT ID NCT06298630
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Nanfang Hospital, Southern Medical University
ACTIVE_NOT_RECRUITINGGuangzhou, Guangdong, 510006, China
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No. 923 Hospital of Joint Support Force of Chinese People 's Liberation Army Hospital
NOT_YET_RECRUITINGNanning, Guangxi, 530021, China
Contact
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Shenzhen Children 's Hospital
NOT_YET_RECRUITINGShenzhen, Guangdong, 510006, China
Contact
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Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
NOT_YET_RECRUITINGGuangzhou, Guangdong, 510006, China
Contact
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The First Affiliated Hospital of GUANGXI MEDICAL UNIVERSITY
RECRUITINGGuangxi, 530021, China
Contact
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Tianjin Institute of Hematology
RECRUITINGTianjin, Tianjin Municipality, 300000, China
Contact
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Xiangya Hospital of Central South University
RECRUITINGChangsha, Hunan, 510510, China
Contact
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Talking therapy could help thalassemia patients take their meds
- Drug cocktail may cut transfusions for kids with thalassemia
- Scientists track lifespan of transfused blood in sickle cell kids
- New transplant approach could help more patients with blood disorders
- One-Shot gene fix for blood disorder enters human testing
- CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients