Fanconi anemia
MONDO:0019391Fanconi anemia (FA) is a hereditary DNA repair disorder characterized by progressive pancytopenia with bone marrow failure, variable congenital malformations and predisposition to develop hematological or solid tumors.
Also known as: Fanconi anemia, Fanconi pancytopenia, Fanconi's anemia, Panmyelopathy, Fanconi, pancytopenia, congenital, primary erythroid hypoplasia
54 clinical trials for this condition and its sub-types, 29 tagged with Fanconi anemia itself.
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Sub-types of Fanconi anemia
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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 2 • Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Prevention
Last updated Jun 27, 2026 12:10 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 1/2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 26, 2026 16:06 UTC