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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Can a Two-Drug immune attack shrink Hard-to-Treat pancreatic tumors?
Disease control Not yet recruitingThis phase 2 trial is testing whether combining two immunotherapy drugs—vilastobart and retifanlimab—can shrink tumors in people with metastatic pancreatic cancer that has a BRCA1, BRCA2, or PALB2 gene mutation. Participants receive both drugs by IV infusion over several months. …
Phase 2 • Sponsor: Massachusetts General Hospital • Aim: Disease control
Last updated Aug 16, 2026 00:00 UTC
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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 1/2 • Sponsor: Agnieszka Czechowicz • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Could vitamin c tame anemia? early trial tests high doses in rare blood disorders
Disease control Not yet recruitingThis early-stage study tests whether high-dose Vitamin C is safe for people with two rare inherited blood disorders—Pyruvate Kinase Deficiency (PKD) and a form of G6PD deficiency. Both conditions cause red blood cells to break down too quickly, leading to anemia. The study will e…
Phase 1 • Sponsor: University of Utah • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
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Could your own stem cells fight this rare brain disorder?
Disease control Not yet recruitingThis study tests whether a person's own stem cells, processed and given by IV, can safely help with multiple system atrophy (MSA) — a rare, worsening brain disease that affects movement and automatic body functions like blood pressure. Fifty adults aged 35 to 65 will receive eith…
Phase 2 • Sponsor: Biocells Medical • Aim: Disease control
Last updated Jun 27, 2026 11:01 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 2 • Sponsor: EuroBloodNet Association • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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Smartwatch signals may flag anemia, no needle needed
Diagnosis Not yet recruitingAnemia often goes unnoticed because diagnosis requires a blood draw. This study tests whether a wrist-worn smartwatch can identify people likely to have anemia by tracking blood flow, heart rate, oxygen, and movement. About 400 adults, with and without anemia, will wear the watch…
Sponsor: Peking Union Medical College Hospital • Aim: Diagnosis
Last updated Sep 03, 2026 00:00 UTC
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Could a painless light sensor replace the needle for anemia screening?
Diagnosis Not yet recruitingThis study tests whether a non-invasive device that uses light to measure hemoglobin through the skin can accurately detect anemia. Researchers will compare its readings with standard blood tests in 150 adults in Jakarta. If the device proves reliable, it could offer a quick, pai…
Sponsor: Tarumanagara University • Aim: Diagnosis
Last updated Jul 17, 2026 00:00 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…
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…
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 Sep 21, 2026 22:00 UTC