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
57 clinical trials for this condition and its sub-types.
Follow this condition — get notified about new trialsSub-types
Broader categories
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Blood transfusion thresholds tested to save elderly surgical patients
Disease control CompletedThis study tests whether giving blood transfusions more liberally (when hemoglobin drops to 9 g/dL) versus more restrictively (when it drops to 7.5 g/dL) reduces death and serious complications like heart attack, stroke, or kidney injury in patients aged 70 and older undergoing n…
Phase: PHASE3 • Sponsor: Johann Wolfgang Goethe University Hospital • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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New hope for rare blood disorder: experimental pill reduces need for transfusions
Disease control CompletedThis study tested a drug called bitopertin in 15 adults with Diamond-Blackfan anemia (DBA), a rare inherited condition that causes severe anemia. Participants took the pill daily for 8 months, with doses possibly increasing over time. The goal was to see if the drug could raise h…
Phase: PHASE1, PHASE2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Disease control
Last updated Jul 02, 2026 00:00 UTC
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New transplant cocktail aims to cut complications in bone marrow failure
Disease control CompletedThis phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…
Phase: PHASE2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 11:00 UTC
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One donor, two transplants: could this end lifelong Anti-Rejection drugs?
Disease control CompletedThis pilot study tested a combined bone marrow and kidney transplant from a partially matched family donor in 10 people with both a blood disorder and chronic kidney disease. The goal was to treat both conditions at once and possibly reduce the need for lifelong anti-rejection dr…
Phase: NA • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Could a natural compound help prevent cancer in a rare disease?
Prevention CompletedThis study tested whether quercetin, a natural substance found in some foods, could help prevent a type of mouth cancer in people with Fanconi anemia, a rare genetic condition that raises cancer risk. 48 participants took quercetin to see if it reduced certain cell changes linked…
Phase: PHASE2 • Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Prevention
Last updated Jun 27, 2026 12:10 UTC
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Anemia in kids may be tied to bigger hearts, study finds
Knowledge-focused CompletedThis study explored whether low hemoglobin (anemia) is linked to an enlarged heart (cardiomegaly) in children up to 18 years old. Researchers used chest X-rays and echocardiograms to check for heart enlargement in 56 anemic children. The goal was to better understand how anemia a…
Sponsor: Universitas Sumatera Utara • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:00 UTC
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Small step toward gene therapy for rare anemia
Knowledge-focused CompletedThis pilot study tested whether a combination of two drugs (G-CSF and plerixafor) could safely collect enough stem cells from the blood of children with Fanconi anemia for future gene therapy. Only 4 patients took part, and the main goal was to see if the cell collection process …
Phase: PHASE1, PHASE2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 26, 2026 16:06 UTC