THALASSEMIA
Clinical trials for THALASSEMIA explained in plain language.
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One-Shot CRISPR treatment aims to cure blood disorder, end lifelong transfusions
⭐️ CURE ⭐️ CompletedThis study tested a one-time treatment for people with severe beta-thalassemia, a genetic blood disorder that requires regular blood transfusions to survive. Doctors used CRISPR gene-editing technology to modify a patient's own blood stem cells, then gave them back as a transplan…
Matched conditions: THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: ⭐️ CURE ⭐️
Last updated Mar 25, 2026 14:09 UTC
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New sickle cell Drug's heart safety put to the test
Disease control CompletedThis study tested whether a potential new medicine for sickle cell disease, called etavopivat, affects the heart's electrical activity in healthy people. It involved 33 healthy volunteers who received different doses of the drug, a placebo, or an approved comparison drug to check…
Matched conditions: THALASSEMIA
Phase: PHASE1 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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Common supplement tested to ease Heart-Lung strain in blood disorder
Disease control CompletedThis study investigated whether adding the supplement L-glutamine to standard care could lower high blood pressure in the lung arteries of adults with a specific type of thalassemia, a genetic blood disorder. For 60 days, one group took oral L-glutamine while a control group rece…
Matched conditions: THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Mazandaran University of Medical Sciences • Aim: Disease control
Last updated Mar 30, 2026 14:31 UTC
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New pill aims to cut blood transfusions for blood disorder patients
Disease control CompletedThis study tested an oral medication called etavopivat in people with sickle cell disease or thalassemia. The main goals were to see if the drug was safe and if it could help patients need fewer blood transfusions or have higher hemoglobin levels. The trial included 53 participan…
Matched conditions: THALASSEMIA
Phase: PHASE2 • Sponsor: Forma Therapeutics, Inc. • Aim: Disease control
Last updated Mar 25, 2026 14:08 UTC
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One donor, two transplants: experimental procedure aims to cure blood cancer and fix kidneys
Disease control CompletedThis pilot study tested a combined transplant from a partially matched family donor. It aimed to treat patients who had both a serious blood disorder (like leukemia) and advanced kidney disease at the same time. The goal was to cure the blood disorder and restore kidney function …
Matched conditions: THALASSEMIA
Phase: NA • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Mar 24, 2026 12:02 UTC
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First test of potential blood disorder drug in chinese volunteers
Knowledge-focused CompletedThis early study tested a new medicine called etavopivat, which is being developed for sickle cell disease and thalassemia. It involved 24 healthy Chinese volunteers to see how their bodies process a single dose of the drug and to check for any side effects. The main goal was to …
Matched conditions: THALASSEMIA
Phase: PHASE1 • Sponsor: Novo Nordisk A/S • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:28 UTC
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Researchers map hidden heart iron risk in transfusion patients
Knowledge-focused CompletedThis study aimed to understand how often dangerous iron builds up in the heart of patients who need frequent blood transfusions for conditions like thalassemia, sickle cell disease, or myelodysplasia. Researchers observed 110 patients across 14 centers, using heart and liver MRI …
Matched conditions: THALASSEMIA
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Mar 24, 2026 12:02 UTC