Familial hemolytic anemia
MONDO:0003689A congenital hemolytic anemia caused by defects of the erythrocyte membrane, enzyme deficiencies, or hemoglobinopathies.
Also known as: congenital hemolytic anemia, hereditary hemolytic anemia, anaemia hemolytic congenital, anemia hemolytic congenital
36 clinical trials for this condition and its sub-types, 4 tagged with Familial hemolytic anemia itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Familial hemolytic anemia
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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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Congenital dyserythropoietic anemia 4 trials · 5 incl. sub-types
9 sub-types
- Congenital dyserythropoietic anemia type 1 1 trial Sub-types →
- Congenital dyserythropoietic anemia type 2 1 trial
- Anemia, congenital dyserythropoietic, type IIIb, autosomal recessive 0 trials
- X-linked dyserythropoetic anemia with abnormal platelets and neutropenia 0 trials
- Anemia, congenital dyserythropoietic, type IVb 0 trials
- Congenital dyserythropoietic anemia type 3 0 trials
- Congenital dyserythropoietic anemia type 4 0 trials
- Pancreatic insufficiency-anemia-hyperostosis syndrome 0 trials
- Thrombocytopenia with congenital dyserythropoietic anemia 0 trials
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Abetalipoproteinemia 2 trials
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Cutaneous porphyria 2 trials
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Hereditary spherocytosis 2 trials
5 sub-types
- Hereditary spherocytosis type 1 0 trials
- Hereditary spherocytosis type 2 0 trials
- Hereditary spherocytosis type 3 0 trials
- Hereditary spherocytosis type 4 0 trials
- Hereditary spherocytosis type 5 0 trials
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Southeast Asian ovalocytosis 2 trials
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Glycogen storage disease VII 1 trial
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Rh deficiency syndrome 0 trials
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X-linked congenital hemolytic anemia 0 trials
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Cryohydrocytosis 0 trials
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Elliptocytosis 1 0 trials
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Elliptocytosis 2 0 trials
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Familial pseudohyperkalemia 0 trials
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Primary CD59 deficiency 0 trials
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Triosephosphate isomerase deficiency 0 trials
Most studied deeper sub-types
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Can a milder transplant cure sickle cell and thalassemia in adults?
Cure OngoingResearchers are testing a stem cell transplant that uses low-dose radiation and two immune-suppressing drugs instead of high-dose chemotherapy. The goal is to cure severe sickle cell disease and beta-thalassemia in adults up to age 65 who have a matched sibling donor. Participant…
Phase 1/2 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Cure
Last updated Sep 11, 2026 00:00 UTC
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Could a daily pill help kids with rare blood disorder?
Disease control OngoingThis study tests a drug called mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic condition that causes red blood cells to break down too quickly, leading to anemia. The trial compares mitapivat to a placebo to see if it can raise hemoglobin levels…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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Could a pill replace blood transfusions for kids with rare anemia?
Disease control OngoingThis phase 3 trial tests the drug mitapivat in children aged 1 to 18 with pyruvate kinase deficiency, a rare genetic disorder that causes red blood cells to break down too quickly. These children need regular blood transfusions. The study compares mitapivat to a placebo to see if…
Phase 3 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 29, 2026 00:00 UTC
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Lifeline drug mitapivat keeps flowing for rare blood disorder patients
Disease control By invitation onlyThis study offers continued access to the drug mitapivat for adults with pyruvate kinase deficiency who completed an earlier Agios-sponsored trial and cannot get the drug commercially. Only 6 participants are enrolled, and the main goal is to monitor side effects. The study does …
Phase 4 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 14, 2026 00:00 UTC
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Could an Iron-Removing pill protect the brain after a burst aneurysm?
Disease control OngoingThis study tests whether the drug deferiprone can safely lower iron levels in the brain after a bleeding aneurysm. About 66 adults who had a ruptured aneurysm will receive either deferiprone or a placebo for 14 days. Researchers will measure iron in spinal fluid, brain MRI scans,…
Phase 1/2 • Sponsor: Duke University • Aim: Disease control
Last updated Jul 22, 2026 00:00 UTC
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New drug combo aims to make bone marrow transplants safer for kids
Disease control OngoingThis pilot study tests a fludarabine-based drug regimen to prepare children with bone marrow failure syndromes for a bone marrow transplant from a matched sibling donor. The goal is to help the donor cells successfully take root while reducing serious side effects. The study incl…
Early phase 1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 12:39 UTC
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Gene therapy offers hope for rare blood disorder
Disease control PausedThis study tests a gene therapy for people with pyruvate kinase deficiency, a rare blood disorder causing severe anemia. Ten participants will receive their own blood stem cells modified with a healthy gene to help produce normal red blood cells. The goal is to raise hemoglobin l…
Phase 2 • Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:08 UTC
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New blood treatment could block zika in transfusions
Disease control OngoingThis study tests a special treatment for red blood cells that aims to kill the Zika virus, making transfusions safer. About 692 people who need blood transfusions will receive either treated or standard blood. The goal is to see if the treated blood works just as well and is safe…
Phase 3 • Sponsor: Cerus Corporation • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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Global registry launches to unlock secrets of rare blood disorder
Knowledge-focused OngoingThis study is a global registry that will follow about 500 people with pyruvate kinase (PK) deficiency, a rare inherited anemia, for up to 9 years. Researchers will collect medical data to better understand the disease's natural history, treatments, and complications. No new drug…
Sponsor: Agios Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Aug 29, 2026 00:00 UTC