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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New hope for anemia patients: drug may cut need for blood transfusions
Disease control OngoingThis study tests a drug called luspatercept in people with anemia caused by myelodysplastic syndromes (MDS) or beta-thalassemia who need regular blood transfusions. The goal is to see if the drug is safe and can reduce the number of transfusions needed. About 85 participants in I…
Phase 4 • Sponsor: Bristol-Myers Squibb • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
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CRISPR gene therapy: a Long-Term safety check for blood disease patients
Disease control By invitation onlyThis study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the…
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Aug 13, 2026 00:00 UTC
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CRISPR stem cell therapy shows promise for kids with sickle cell
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive the…
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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CRISPR gene therapy aims to free kids from lifelong blood transfusions
Disease control OngoingThis phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the…
Phase 3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jul 03, 2026 00:00 UTC
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New pill could slash sickle cell pain crises
Disease control OngoingThis large Phase 3 trial is testing an oral drug called etavopivat in 450 people with sickle cell disease. The goal is to see if taking a pill once daily can raise hemoglobin levels and reduce the number of painful vaso-occlusive crises compared to a placebo. Participants must ha…
Phase 3 • Sponsor: Forma Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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New transplant approach offers hope for sickle cell patients without a perfect donor match
Disease control OngoingThis early-phase study tests a new type of stem cell transplant for people with severe sickle cell disease who don't have a fully matched sibling donor. It uses a half-matched family donor and a milder chemotherapy regimen to prepare the body, along with removing certain immune c…
Phase 1 • Sponsor: City of Hope Medical Center • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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New transplant method aims to cut dangerous immune reaction in half-matched donors
Disease control OngoingThis study tests a stem cell transplant method that removes certain immune cells from the donor's blood to lower the chance of graft-versus-host disease (GvHD), a serious complication. It involves 3 patients with blood cancers or non-cancerous blood disorders who lack a perfectly…
Sponsor: Baylor College of Medicine • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC