Inherited hemoglobinopathy
MONDO:0019050An inherited disorder characterized by structural alterations of a globin chain within the hemoglobin molecule.
Also known as: Hemoglobinopathies / iron metabolism, hereditary hemoglobinopathy, hemoglobinopathy
444 clinical trials for this condition and its sub-types, 38 tagged with Inherited hemoglobinopathy itself.
Follow this condition to get notified about new trialsWhere it sits in the disease tree
Browse by category →Sub-types of Inherited hemoglobinopathy
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Sickle cell disease 343 trials
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Thalassemia 49 trials · 129 incl. sub-types
2 sub-types
- Beta thalassemia 73 trials · 99 incl. sub-types Sub-types →
- Alpha thalassemia spectrum 11 trials · 12 incl. sub-types Sub-types →
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Beta-thalassemia and related diseases 0 trials · 99 incl. sub-types
7 sub-types
- Beta thalassemia 73 trials · 99 incl. sub-types Sub-types →
- Hemoglobin E-beta-thalassemia syndrome 5 trials
- Beta-thalassemia-X-linked thrombocytopenia syndrome 0 trials
- Delta-beta-thalassemia 0 trials
- Hemoglobin C-beta-thalassemia syndrome 0 trials
- Hemoglobin Lepore-beta-thalassemia syndrome 0 trials
- Hereditary persistence of fetal hemoglobin-beta-thalassemia syndrome 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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Sickle cell-beta-thalassemia disease syndrome 5 trials · 9 incl. sub-types
2 sub-types
- Sickle cell-beta zero-thalassemia 6 trials
- Sickle cell-beta plus-thalassemia 5 trials
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Hemoglobin C disease 2 trials
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Unstable hemoglobin disease 1 trial
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Hereditary methemoglobinemia 0 trials · 1 incl. sub-types
5 sub-types
- Methemoglobinemia type 4 1 trial
- Hemoglobin M disease 0 trials
- Methemoglobin reductase deficiency 0 trials
- Methemoglobinemia due to deficiency of methemoglobin reductase 0 trials
- Methemoglobinemia, alpha type 0 trials
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Hemoglobin D disease 0 trials
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Hemoglobin E disease 0 trials
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Hemoglobinopathy Toms River 0 trials
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Homozygous hemoglobin O Arab disease 0 trials
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Sulfhemoglobinemia, congenital 0 trials
Most studied deeper sub-types
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Can a One-Time gene infusion free patients from transfusions?
Cure Not yet recruitingThis trial tests a gene therapy called YOLT-204 in people aged 14 to 35 with sickle cell disease or transfusion-dependent thalassemia. The goal is to see if a single intravenous dose can safely boost fetal hemoglobin levels, potentially reducing or eliminating the need for blood …
Sponsor: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology • Aim: Cure
Last updated Aug 21, 2026 00:00 UTC
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Selective cell removal may tame stem cell Transplant's dangerous side effect
Disease control Not yet recruitingThis study tests whether removing a specific type of immune cell (naive T cells) from a donor's stem cell graft can prevent graft-versus-host disease (GVHD), a serious complication where donor cells attack the recipient's body. The trial enrolls people with non-malignant blood di…
Phase 2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Jun 27, 2026 14:01 UTC
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New drug YOLT-204 aims to reduce transfusions in blood disorders
Disease control Not yet recruitingThis early-phase trial will test a single dose of a new drug called YOLT-204 in 18 children and teens with transfusion-dependent β-thalassemia or sickle cell disease. The goal is to see if it is safe and can raise fetal hemoglobin levels enough to reduce the need for blood transf…
Early phase 1 • Sponsor: Guangzhou Women and Children's Medical Center • Aim: Disease control
Last updated Jun 27, 2026 07:51 UTC