Plain-English translation of NCT06465550 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This trial is testing BD211, a new gene therapy treatment designed to help the body produce healthy red blood cells in patients with β-thalassemia, a serious inherited blood disorder. Currently, people with this condition need regular blood transfusions to survive. The study will give one infusion of specially modified cells to about 9 patients and follow them for 18 months to see if the treatment is safe and whether it reduces or eliminates their need for transfusions.
Children and young adults with severe β-thalassemia need frequent blood transfusions, which can cause serious side effects and carry risks. This medication offers a potential one-time cure by giving the body the ability to make its own healthy blood cells, which could free patients from lifelong transfusion dependence.
You likely qualify if…
You likely don't qualify if…
You will receive one infusion of the modified cells through an IV line. After the infusion, you will need to visit the clinic regularly over 18 months for blood tests, physical exams, and other safety checks to see how well the treatment is working and to monitor for any side effects. You will also be asked to keep detailed records and stick to your current iron management plan during the study.
AI-generated summary from trial data · Aug 24, 2026 · Not medical advice
China