Gene therapy aims to free Beta-Thalassemia patients from lifelong transfusions

NCT ID NCT07680803

Recruiting now Disease control Sponsor: Fondazione Telethon Source: ClinicalTrials.gov ↗

First seen Jul 02, 2026 · Last updated Jul 28, 2026 · Updated 2 times

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.

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Conditions

The condition(s) this trial relates to.

beta-thalassemia major

As listed by the trial registrant

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

TRANSFUSION-DEPENDENT BETA-THALASSEMIA

Contacts and locations

Locations

Ospedale Pediatrico Bambino Gesù
RECRUITING

Rome, Lazio, 00165, Italy
Ospedale San Raffaele
RECRUITING

Milan, Lombardy, 20132, Italy

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