BETA-THALASSEMIA MAJOR
Clinical trials for BETA-THALASSEMIA MAJOR explained in plain language.
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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…
Matched conditions: BETA-THALASSEMIA MAJOR
Phase: PHASE2 • Sponsor: New York Medical College • Aim: ⭐️ CURE ⭐️
Last updated Jul 29, 2026 00:00 UTC
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Mismatched donor stem cell transplant could widen treatment access for sickle cell disease
Disease control Recruiting nowThis study tests a stem cell transplant using cells from mismatched unrelated or partially matched family donors for people with severe sickle cell disease and other transfusion-dependent anemias. The approach removes certain immune cells (T-cells) from the donated stem cells to …
Matched conditions: BETA-THALASSEMIA MAJOR
Phase: PHASE1, PHASE2 • Sponsor: Paul Szabolcs • Aim: Disease control
Last updated Aug 15, 2026 00:00 UTC
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Old drugs, new hope: could a thalidomide combo free thalassemia patients from transfusions?
Disease control Recruiting nowThis study tests whether a combination of two drugs, thalidomide and hydroxyurea, can safely boost fetal hemoglobin levels in people with beta thalassemia major. The goal is to improve overall hemoglobin levels and reduce the need for regular blood transfusions. The trial include…
Matched conditions: BETA-THALASSEMIA MAJOR
Phase: NA • Sponsor: Riphah International University • Aim: Disease control
Last updated Aug 08, 2026 00:03 UTC