Hemoglobin E-beta-thalassemia syndrome
MONDO:0016491Hemoglobin E - beta-thalassemia (HbE - BT) is a form of beta-thalassemia that results in a mild to severe clinical presentation ranging from a condition indistinguishable from beta-thalassemia major to a mild form of beta-thalassemia intermedia.
Also known as: E-beta-thalassemia, HbE-beta-thalassemia syndrome
27 clinical trials for this condition and its sub-types, 5 tagged with Hemoglobin E-beta-thalassemia syndrome itself.
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New drug hopes to ease transfusion burden for kids with blood disorder
Disease control Recruiting nowThis study tests a drug called luspatercept in children aged 6 to 18 with beta-thalassemia, a blood disorder that often requires regular transfusions. The goal is to see if the drug is safe and can reduce the need for transfusions or raise hemoglobin levels. The trial is still re…
Phase 2 • Sponsor: Celgene • Aim: Disease control
Last updated Jul 12, 2026 00:00 UTC
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New pill could cut transfusions for thalassemia patients
Disease control Recruiting nowThis Phase 2 study tests an oral drug called AND017 in 64 people with β-thalassemia, a blood disorder that often requires regular transfusions. Half the participants get the drug, half get a placebo, and everyone continues standard care. The goal is to see if AND017 is safe and c…
Phase 2 • Sponsor: Kind Pharmaceuticals LLC • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Leftover bone marrow could unlock better gene therapies for sickle cell disease
Knowledge-focused Recruiting nowThis study collects bone marrow that is normally thrown away during surgery from people with and without blood disorders like sickle cell disease. Researchers will use these samples in the lab to learn how to better manipulate stem cells for gene therapy and to study cell health.…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:29 UTC