Inherited aplastic anemia
MONDO:0001713An inborn condition characterized by deficiencies of red cell precursors that sometimes also includes leukopenia and thrombocytopenia.
Also known as: constitutional aplastic anaemia, constitutional aplastic anemia, hereditary aplastic anaemia, hereditary aplastic anemia, rare constitutional aplastic anaemia, rare constitutional aplastic anemia, congenital aplastic anaemia, congenital aplastic anemia
85 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
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
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A simple brush could spot mouth cancer early in fanconi anaemia patients
Diagnosis Not yet recruitingPeople with Fanconi anaemia have a high risk of developing oral cancer, but standard biopsies are painful and risky due to their genetic sensitivity. This study tests a non-invasive oral brushing technique to detect early cancer cells in 115 patients aged 15 and older. If accurat…
Phase: NA • Sponsor: Institut Jean-Godinot • Aim: Diagnosis
Last updated Jun 27, 2026 07:58 UTC
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New study tests which laser technique clears kidney stones faster
Symptom relief Not yet recruitingThis study compares two ways to break up kidney stones using a laser and a suction device. One method turns stones into dust, the other into small fragments. The goal is to see which leaves fewer stone pieces behind after one month. About 86 adults with small kidney stones will t…
Phase: NA • Sponsor: Bir Hospital • Aim: Symptom relief
Last updated Jul 02, 2026 00:00 UTC
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Could you help scientists unlock genetic cancer secrets?
Knowledge-focused Not yet recruitingThis study screens up to 1,000 people with personal or family histories of certain cancers to see if they qualify for ongoing genetics research at the National Cancer Institute. Participants fill out a 15-20 minute online survey about their health and family history. No treatment…
Sponsor: National Cancer Institute (NCI) • Aim: Knowledge-focused
Last updated Jul 04, 2026 00:00 UTC