Gene therapy aims to free Beta-Thalassemia patients from lifelong transfusions
NCT ID NCT07680803
First seen Jul 02, 2026 · Last updated Jul 02, 2026
Summary
This study tests a single-dose gene therapy for people with transfusion-dependent beta-thalassemia, a blood disorder that requires regular red blood cell transfusions. The therapy uses a patient's own stem cells, modified with a virus to produce healthy hemoglobin, and then infused back. The goal is to see if this approach can allow patients to go without transfusions for at least 12 months. The trial involves 9 participants across two centers in Italy.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
gene therapy using modified stem cells (GLOBE lentiviral vector)
What this could lead to
If successful, this one-time treatment could free people with beta-thalassemia from needing regular blood transfusions.
What could go wrong
This is an early-phase trial with only 9 participants, so results may not apply to everyone. Gene therapy carries risks like infection or immune reactions.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Hydroxyurea vs Metformin for Thalassemia (NCT06606886)
- Mitapivat for Transfusion-Dependent Thalassemia NCT07506863
- Stem Cell Transplant for Blood Disorders - NCT06872333
- GLP-1 Drug for Diabetes in Thalassemia Kids (NCT07370922)
- Hypoxic RBC Storage for Thalassemia Transfusion NCT07055503
- Thalidomide & Hydroxyurea for Thalassemia (NCT07292259)
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ospedale Pediatrico Bambino Gesù
Rome, Lazio, 00165, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Ospedale San Raffaele
Milan, Lombardy, 20132, Italy
Contact Phone: •••-•••-•••• Email: •••••@•••••