BETA-THALASSEMIA MAJOR
Clinical trials for BETA-THALASSEMIA MAJOR explained in plain language.
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New stem cell transplant approach shows promise for blood disorders
Disease control CompletedThis pilot study tested a stem cell transplant from closely matched unrelated donors for 8 patients with severe sickle cell disease or thalassemia major. The donated stem cells were specially processed to remove certain immune cells to reduce the risk of rejection and graft-versu…
Matched conditions: BETA-THALASSEMIA MAJOR
Phase: NA • Sponsor: Timothy Olson • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC
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Gene therapy breakthrough? early trial for blood disorder shows promise
Disease control CompletedThis early-stage study tested a single dose of a gene therapy called CS-101 in 9 people with beta-thalassemia major, a severe blood disorder requiring lifelong transfusions. The main goals were to check safety and see if the treatment helps the body make more healthy hemoglobin. …
Matched conditions: BETA-THALASSEMIA MAJOR
Phase: PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:03 UTC
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Simple blood tests may spot liver damage in thalassemia kids
Diagnosis CompletedThis study looked at 100 children with beta-thalassemia major and iron overload to see if simple blood tests (ferritin and hepcidin) can detect liver scarring (fibrosis) as accurately as other non-invasive methods. Researchers measured these blood markers and compared them to ult…
Matched conditions: BETA-THALASSEMIA MAJOR
Sponsor: Tanta University • Aim: Diagnosis
Last updated Jun 26, 2026 14:15 UTC
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Simple blood test could transform iron monitoring in thalassemia
Knowledge-focused CompletedThis study tested a new, simpler method to measure iron buildup inside red blood cells of people with beta thalassemia major. The goal was to find a cheaper and easier way to monitor iron overload compared to current tests. Researchers analyzed blood samples from 100 patients to …
Matched conditions: BETA-THALASSEMIA MAJOR
Sponsor: Ankara City Hospital Bilkent • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:04 UTC