Congenital anemia
MONDO:0000577Anemia, the cause of which is present at birth.
Also known as: congenital anaemia (disease), congenital anemia, congenital anemia (disease)
67 clinical trials for this condition and its sub-types, 0 tagged with Congenital anemia itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Congenital anemia
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Fanconi anemia 29 trials · 42 incl. sub-types
22 sub-types
- Fanconi anemia complementation group D1 6 trials
- Fanconi anemia complementation group A 4 trials
- Fanconi anemia complementation group E 4 trials
- Fanconi anemia complementation group N 2 trials
- Fanconi anemia, complementation group S 2 trials
- Fanconi anemia complementation group B 0 trials
- Fanconi anemia complementation group C 0 trials
- Fanconi anemia complementation group D2 0 trials
- Fanconi anemia complementation group F 0 trials
- Fanconi anemia complementation group G 0 trials
- Fanconi anemia complementation group I 0 trials
- Fanconi anemia complementation group J 0 trials
- Fanconi anemia complementation group L 0 trials
- Fanconi anemia complementation group O 0 trials
- Fanconi anemia complementation group P 0 trials
- Fanconi anemia complementation group Q 0 trials
- Fanconi anemia complementation group R 0 trials
- Fanconi anemia complementation group T 0 trials
- Fanconi anemia complementation group U 0 trials
- Fanconi anemia complementation group V 0 trials
- Fanconi anemia, complementation group W 0 trials
- Fanconi anemia, complementation group 10 0 trials
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Congenital nonspherocytic hemolytic anemia 1 trial · 12 incl. sub-types
10 sub-types
- Pyruvate kinase deficiency of red cells 10 trials
- Anemia, nonspherocytic hemolytic 0 trials · 2 incl. sub-types Sub-types →
- Hemolytic anemia due to adenylate kinase deficiency 1 trial
- Hemolytic anemia due to pyrimidine 5' nucleotidase deficiency 1 trial
- Gamma-glutamylcysteine synthetase deficiency 0 trials
- Glutathione synthetase deficiency without 5-oxoprolinuria 0 trials
- Hemolytic anemia due to erythrocyte adenosine deaminase overproduction 0 trials
- Hemolytic anemia due to glucophosphate isomerase deficiency 0 trials
- Hemolytic anemia due to glutathione reductase deficiency 0 trials
- Non-spherocytic hemolytic anemia due to hexokinase deficiency 0 trials
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3 sub-types
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2 sub-types
Most studied deeper sub-types
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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 3 • Sponsor: Johann Wolfgang Goethe University Hospital • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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Promising new drug shows hope for rare anemia patients
Disease control CompletedThis study tested a drug called mitapivat (AG-348) in 52 adults with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The goal was to see if different doses of the drug are safe and help control the dis…
Phase 2 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
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New hope for rare blood disorder: Long-Term drug study shows promise
Disease control CompletedThis study looked at the long-term safety and effectiveness of the drug mitapivat in 90 adults with pyruvate kinase deficiency, a rare genetic blood disorder that causes red blood cells to break down too quickly. Participants had already completed earlier studies of mitapivat and…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 07:58 UTC
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Gene therapy offers hope for rare blood disorder
Disease control CompletedThis phase 1 trial tested a gene therapy called RP-L301 in 4 people with pyruvate kinase deficiency, a rare inherited blood disorder that causes severe anemia and often requires frequent blood transfusions. The treatment uses the patient's own blood stem cells, which are modified…
Phase 1 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 26, 2026 13:53 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 2 • Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Prevention
Last updated Jun 27, 2026 12:10 UTC
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Blood cancer enzyme deficiency explored in new study
Knowledge-focused CompletedThis study looked at how often people with certain blood cancers or related conditions have an acquired deficiency of an enzyme called pyruvate kinase, which can cause anemia. Researchers took a single blood sample from 18 participants to measure enzyme activity and check for rel…
Sponsor: Massachusetts General Hospital • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:35 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 1/2 • Sponsor: Assistance Publique - Hôpitaux de Paris • Aim: Knowledge-focused
Last updated Jun 26, 2026 16:06 UTC