Diamond-Blackfan anemia
MONDO:0015253A congenital aregenerative and often macrocytic anemia with erythroblastopenia.
Also known as: Aase syndrome, Aase-Smith II syndrome, Blackfan-Diamond anaemia, Blackfan-Diamond anemia, DBA, Diamond Blackfan Anemia, Diamond-Blackfan anemia, chronic constitutional pure red cell anemia
56 clinical trials for this condition and its sub-types.
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Broader categories
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Could a stem cell shot before birth fix fanconi anemia?
Disease control Not yet recruitingThis early-phase trial tests whether giving healthy stem cells from the mother to a fetus diagnosed with Fanconi anemia can safely improve the baby's blood cell production. The treatment is a one-time injection into the fetus during pregnancy. The study will enroll 12 pregnant wo…
Phase: PHASE1, PHASE2 • Sponsor: Agnieszka Czechowicz • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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New CAR-T therapy targets antibodies blocking Life-Saving transplants
Disease control Not yet recruitingThis early-stage study tests a new treatment using special immune cells (CAR-T cells) designed to reduce harmful antibodies that can prevent or damage bone marrow transplants. About 18 people with blood diseases who have these antibodies will receive the cells in increasing doses…
Phase: PHASE1, PHASE2 • Sponsor: Chang Yingjun • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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New hope for rare anemias: drug may cut transfusions
Disease control Not yet recruitingThis Phase 2 trial tests the drug luspatercept (Reblozyl) in 45 people with rare inherited anemias that affect red blood cells. The goal is to see if it can reduce the need for blood transfusions in those who depend on them, or raise hemoglobin levels in those who don't. Particip…
Phase: PHASE2 • Sponsor: EuroBloodNet Association • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Could a feeding tube after transplant save the gut?
Disease control Not yet recruitingThis study tests whether starting tube feeding right after a stem cell transplant can reduce severe gut graft-versus-host disease (GVHD) in people with blood cancers or disorders. About 112 participants will be randomly assigned to either standard care or early tube feeding. The …
Phase: PHASE2 • Sponsor: University of Nebraska • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC