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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Gene editing offers hope for a One-Time sickle cell cure
Cure CompletedResearchers are testing a one-time gene therapy called exa-cel for people with severe sickle cell disease. The treatment uses CRISPR-Cas9 to edit a patient's own blood stem cells, which are then infused back after chemotherapy. The trial measures whether participants remain free …
Phase 2/3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Cure
Last updated Sep 18, 2026 00:00 UTC
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Gentler transplant shows promise for kids with blood diseases
Disease control CompletedThis study tests a milder chemotherapy and immune-suppressing regimen before a stem cell transplant for children and young adults with non-malignant blood disorders like sickle cell disease or immune deficiencies. The goal is to safely achieve donor cell engraftment with fewer si…
Phase 2 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jul 23, 2026 00:00 UTC
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New pill could cut blood transfusions for blood disorders
Disease control CompletedThis Phase 2 study tested an experimental drug called etavopivat in 53 people with thalassemia or sickle cell disease. The goal was to see if the pill could safely reduce the number of red blood cell transfusions needed and raise hemoglobin levels. Participants took 400 mg of eta…
Phase 2 • Sponsor: Forma Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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CRISPR gene editing offers hope for thalassemia patients to ditch transfusions
Disease control CompletedThis study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long th…
Phase 2/3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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New chemo combo may lower death risk in child stem cell transplants
Disease control CompletedThis study tested two different chemotherapy drugs (Treosulfan and Busulfan) given before a stem cell transplant in 106 children with serious non-cancer diseases like immune disorders, metabolic diseases, blood disorders, and bone marrow failure. The goal was to see which drug le…
Phase 2 • Sponsor: medac GmbH • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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Could carbon monoxide be a medicine? new trial for sickle cell
Symptom relief CompletedThis study tests a new liquid drug called HBI-002, which contains carbon monoxide, in 9 people with sickle cell disease aged 14 to 55. Participants take the drug daily for 14 days to see if it is safe and helps with symptoms. The goal is to find a better way to manage this painfu…
Phase 2 • Sponsor: Hillhurst Biopharmaceuticals, Inc. • Aim: Symptom relief
Last updated Aug 08, 2026 00:03 UTC
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New hope for sickle cell pain: canakinumab shows promise in kids
Symptom relief CompletedThis study tested a drug called canakinumab (ACZ885) in 49 children and young adults aged 8-20 with sickle cell anemia. The goal was to see if it could reduce daily pain compared to a placebo. Participants rated their pain daily using a 0-10 scale, and researchers measured change…
Phase 2 • Sponsor: Novartis Pharmaceuticals • Aim: Symptom relief
Last updated Jun 27, 2026 08:10 UTC
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8,400 screened in bangladesh to uncover hidden blood disorders
Knowledge-focused CompletedThis study screened 8,400 people living in Chattogram, Bangladesh to find out how common abnormal hemoglobin types are. Researchers used a blood test called HbA1c capillary electrophoresis to detect conditions like HbE trait. The goal was to understand the prevalence of these inh…
Sponsor: Bangladesh Bioscience Research Group • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:06 UTC
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Blood disorder survey in bangladesh reveals hidden genetic burden
Knowledge-focused CompletedThis completed study looked at how often hemoglobin disorders (like thalassemia and sickle cell disease) occur in people visiting a hospital in Chattogram, Bangladesh. Researchers tested blood samples from 760 volunteers using advanced methods to find different hemoglobin types. …
Sponsor: Bangladesh Bioscience Research Group • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:54 UTC
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Blood disorder survey reveals regional patterns in pakistan
Knowledge-focused CompletedThis completed study checked how often inherited blood disorders (hemoglobinopathies) occur in people from Mardan, Pakistan, and nearby areas. Researchers analyzed blood samples from 839 people using special lab tests. The goal was to gather local data to help improve public heal…
Sponsor: Bacha Khan Medical College • Aim: Knowledge-focused
Last updated Jun 26, 2026 17:45 UTC