Testing AND017 for beta-thalassemia
Treatments studied
Part of Blood & lymphatic clinical trials.
This trial tests an oral medication called AND017 for people with beta-thalassemia. It aims to see if the drug can safely help reduce the need for blood transfusions or improve hemoglobin levels.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of beta-thalassemia or a related form (like hemoglobin E/beta-thalassemia).
- You need to be either transfusion-dependent (getting 6–20 units of blood in the last 6 months, with no break longer than 5 weeks) or non-transfusion-dependent (fewer than 6 units in 6 months and no transfusion in the last 4 weeks).
- Your overall health should be good (ECOG score 0–1, meaning you can do most daily activities).
- If you are non-transfusion-dependent, your hemoglobin must be 10.0 g/dL or lower, and confirmed by two tests at least a week apart.
- You cannot have other causes of anemia, active infections, eye problems needing treatment, recent serious bleeding, uncontrolled high blood pressure, heart failure, or a history of stroke or seizures.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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This trial tests a new gene therapy (BD211) for children and teens with severe beta-thalassemia who need regular blood transfusions. The treatment uses your own blood stem cells, modified with a working gene, to help your body make healthy red blood cells and reduce the need for transfusions.
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