Beta-thalassemia intermedia
MONDO:0016487Beta-thalassemia (BT) intermedia is a form of BT characterized by mild to moderate anemia which does not or only occasionally requires transfusion.
34 clinical trials for this condition and its sub-types, 9 tagged with Beta-thalassemia intermedia itself.
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A drug that protects the liver may make stem cell transplants safer for children with sickle cell disease
Cure Recruiting nowThis trial tests whether giving defibrotide before and after a stem cell transplant can prevent a serious liver condition called sinusoidal obstructive syndrome (SOS) in children and young adults with sickle cell disease or beta thalassemia. Participants receive defibrotide start…
Phase 2 • Sponsor: New York Medical College • Aim: Cure
Last updated Jul 29, 2026 00:00 UTC
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New pill could help kids with thalassemia fight anemia without transfusions
Disease control Recruiting nowThis Phase 3 trial tests whether the drug mitapivat can improve anemia in children aged 1 to 17 with alpha- or beta-thalassemia who do not need regular blood transfusions. About 45 participants will receive either mitapivat or a placebo for 24 weeks. The main goal is to see if mi…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC