BETA-THALASSEMIA MAJOR
Clinical trials for BETA-THALASSEMIA MAJOR explained in plain language.
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Gene therapy breakthrough offers hope for blood disorder patients
Disease control CompletedThis early-stage trial tested a single dose of CS-101, a gene therapy, 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 produce healthy red blood cells. Wh…
Matched conditions: BETA-THALASSEMIA MAJOR
Phase: PHASE1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated May 26, 2026 09:26 UTC
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New way to track iron overload in thalassemia patients?
Knowledge-focused CompletedThis study looked at 100 people with beta thalassemia major, a serious blood disorder requiring frequent blood transfusions. Transfusions can cause dangerous iron buildup in the body. Currently, doctors use a blood test called ferritin to monitor iron levels, but ferritin can be …
Matched conditions: BETA-THALASSEMIA MAJOR
Sponsor: Ankara City Hospital Bilkent • Aim: Knowledge-focused
Last updated May 22, 2026 14:04 UTC
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New study seeks best way to spot liver damage in thalassemia kids
Knowledge-focused CompletedThis study looked at 100 children with beta-thalassemia major and iron overload to see which tests best detect liver scarring. Researchers compared blood markers (ferritin, hepcidin) and non-invasive scores (APRI, FIB-4) against a special ultrasound (FibroScan). The goal was to f…
Matched conditions: BETA-THALASSEMIA MAJOR
Sponsor: Tanta University • Aim: Knowledge-focused
Last updated May 18, 2026 12:07 UTC