Gene therapy breakthrough offers hope for Transfusion-Free life in thalassemia
NCT ID NCT07489196
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a single dose of CS-101, a gene therapy, in 20 people aged 12-35 with β-thalassemia major, a severe blood disorder requiring regular transfusions. The goal is to see if it is safe and can help patients produce enough healthy red blood cells to stop needing transfusions for at least a year. Participants will be monitored for side effects and blood cell recovery.
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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
Locations
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Children's Hospital of Fudan University
Shanghai, Shanghai Municipality, China
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Ruijin Hospital Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, China
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The First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New stem cell transplant approach shows promise for blood disorders
- Gene-Editing breakthrough offers hope for blood disorder patients
- Gentler transplant shows promise for kids with blood diseases
- New study aims to boost growth and health in kids with thalassemia