Gene therapy offers new hope for sickle cell patients
NCT ID NCT03964792
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested a gene therapy for sickle cell disease in 6 patients aged 12-20. Doctors took the patients' own blood stem cells, added a working gene to help produce healthy red blood cells, and infused them back after chemotherapy. The goal was to see if the treatment is safe and can reduce severe pain crises and other complications.
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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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Department of Biotherapy, Necker-Enfants Malades Hospital
Paris, 75015, France
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Other studies related to the condition(s) this trial covers.
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