Plain-English translation of NCT02906202 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Beta-thalassemia research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This trial studied LentiGlobin BB305, a gene therapy designed to treat transfusion-dependent beta-thalassemia, a serious blood disorder that requires frequent blood transfusions. Researchers tested whether the medication—which involves using a patient's own blood stem cells that were genetically modified in the lab—could help patients produce more of their own healthy blood cells. The goal was to reduce or eliminate the need for regular transfusions.
People with transfusion-dependent beta-thalassemia need frequent blood transfusions to survive, which can lead to serious complications from iron buildup in the body. This trial was designed to determine whether this new gene therapy approach could offer patients a potential cure or long-term improvement without requiring a matched bone marrow donor.
Participants received chemotherapy (busulfan) to prepare their body for the new cells, then underwent a single intravenous infusion of their own genetically modified stem cells. The stem cells were collected from each participant, modified in a laboratory to produce healthy hemoglobin, and then returned to the patient. Participants were monitored over time to assess whether the treatment worked and was safe.
AI-generated summary from trial data · Aug 22, 2026 · Not medical advice
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