Β -THALASSEMIA
Clinical trials for Β -THALASSEMIA explained in plain language.
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New pill may cut transfusions for blood disorders
Disease control CompletedThis study tested an experimental drug called etavopivat in 53 people with sickle cell disease or thalassemia. The goal was to see if the drug could safely reduce the need for red blood cell transfusions and increase hemoglobin levels. Participants took the drug by mouth, and res…
Matched conditions: Β -THALASSEMIA
Phase: PHASE2 • Sponsor: Forma Therapeutics, Inc. • Aim: Disease control
Last updated May 22, 2026 13:50 UTC
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One donor, two transplants: new hope for patients with blood cancer and kidney failure
Disease control CompletedThis study tested a combined bone marrow and kidney transplant from a partially matched family donor for 10 people with both a serious blood disorder and chronic kidney disease. The goal was to treat both conditions with one donor, potentially reducing the need for lifelong anti-…
Matched conditions: Β -THALASSEMIA
Phase: NA • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated May 18, 2026 12:01 UTC
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CRISPR gene editing frees thalassemia patients from lifelong transfusions
Disease control CompletedThis study tested a one-time treatment using CRISPR gene editing to fix a patient's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the modified cells after a short course of chemotherapy. …
Matched conditions: Β -THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated May 18, 2026 12:00 UTC
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Glutamine shows promise for thalassemia lung pressure
Disease control CompletedThis small study tested whether taking L-glutamine for 60 days could reduce high blood pressure in the lungs of adults with non-transfusion-dependent thalassemia. Eight participants were split into two groups: one received glutamine plus standard care, the other standard care alo…
Matched conditions: Β -THALASSEMIA
Phase: PHASE2, PHASE3 • Sponsor: Mazandaran University of Medical Sciences • Aim: Disease control
Last updated May 11, 2026 20:37 UTC
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Heart iron danger measured in blood disorder patients
Knowledge-focused CompletedThis study looked at how often heart iron overload happens in people with thalassemia, sickle cell disease, or myelodysplasia who receive many blood transfusions. Researchers used MRI scans to measure iron levels in the heart and liver of 110 participants. The goal was to better …
Matched conditions: Β -THALASSEMIA
Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated May 26, 2026 05:23 UTC
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Heart check for new sickle cell drug: healthy volunteers test safety
Knowledge-focused CompletedThis study checked whether etavopivat, a new medicine for sickle cell disease, changes the heart's electrical activity. Thirty-three healthy adults received different doses of the drug, a placebo, or a standard heart medication. The goal was to see if the drug causes any heart rh…
Matched conditions: Β -THALASSEMIA
Phase: PHASE1 • Sponsor: Novo Nordisk A/S • Aim: Knowledge-focused
Last updated May 22, 2026 14:02 UTC
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First look: new blood disorder drug tested in healthy volunteers
Knowledge-focused CompletedThis early-stage study tested a single dose of a new oral medicine called etavopivat in 24 healthy Chinese volunteers. The main goal was to see how the drug moves through and is processed by the body (pharmacokinetics) and to check its initial safety and tolerability. The results…
Matched conditions: Β -THALASSEMIA
Phase: PHASE1 • Sponsor: Novo Nordisk A/S • Aim: Knowledge-focused
Last updated Apr 09, 2026 19:34 UTC