Anemia means your blood doesn't carry enough oxygen, usually because of low red blood cell count or low hemoglobin. Causes range from iron deficiency to chronic kidney disease to inherited conditions like sickle cell disease and thalassemia. Treatment depends entirely on the underlying cause — iron supplements for deficiency, erythropoiesis-stimulating agents for kidney disease, transfusions or newer targeted drugs for inherited forms.
What's actually going on in research
Trials are testing gene therapies for sickle cell disease and beta-thalassemia, small molecules that boost hemoglobin production, drugs that mimic or improve on erythropoietin, and iron formulations that work faster or cause fewer side effects. For inherited anemias, researchers are moving beyond managing symptoms toward correcting the genetic defect itself.
Gene therapy for inherited anemias
Exagamglogene autotemcel and lovotibeglogene autotemcel are FDA-approved gene therapies for sickle cell disease. Additional gene therapies and gene-editing approaches are in trials for sickle cell and beta-thalassemia.
Hemoglobin induction drugs
Voxelotor increased hemoglobin in sickle cell disease before being withdrawn for safety concerns. Other drugs targeting hemoglobin polymerization or fetal hemoglobin induction remain in development.
Improved iron therapies
New intravenous iron formulations aim to replenish stores faster with fewer infusions. Oral iron products with better absorption or fewer gastrointestinal side effects are also being tested.
What to know before you search
Eligibility typically depends on anemia type and severity, transfusion frequency, prior treatments, and whether the anemia is from an inherited condition or secondary to another disease.
What types of trials are currently open
- Gene therapy trials — Testing one-time treatments that modify a patient's own blood stem cells to produce healthy hemoglobin. Typically for sickle cell disease or beta-thalassemia.
- Drug treatment trials — Testing pills or infusions that boost red blood cell production, increase hemoglobin levels, or reduce complications from inherited anemias.
- Iron formulation trials — Comparing new oral or intravenous iron products to see if they restore iron stores faster or with fewer side effects.
- Transfusion reduction trials — Testing whether new drugs can reduce or eliminate the need for regular blood transfusions in people with inherited anemias.
- Registry studies — Following people with specific types of anemia over time to understand disease progression, treatment patterns, and long-term outcomes.
Recently added Anemia trials
A Phase 3 Evaluation of Sutacimig in Glanzmann Thrombasthenia
Glanzmann thrombasthenia (GT) is a serious inherited bleeding disorder. In GT, platelets, the blood cells that normally stick together at the site of an injury to stop bleeding, are unable to properly do so. As a result, bleeding does not stop as it should. Bleeding in GT often starts without warning, can happen frequently, and can be severe. It most commonly affects the nose, mouth and gums, gastrointestinal tract, and skin, and many women and girls with GT experience heavy menstrual bleeding. Over time, bleeding can lead to iron deficiency and anemia. Some bleeding episodes require emergency care, hospitalization, transfusion, or surgery, and some can be life-threatening. Sutacimig is an investigational medicine given as an injection under the skin (subcutaneous injection). It is designed to help platelets work at the site of bleeding despite the underlying defect in GT. It is intended to be given regularly to help prevent bleeding (prophylaxis), rather than only to treat bleeding after it starts. The purpose of this study is to find out whether sutacimig, given as prophylaxis, reduces the number of treated bleeding episodes in people with GT compared with no prophylaxis, and to evaluate its safety and tolerability.
Aplastic Anemia Life-Course Cohort Study
To establish a longitudinal cohort spanning the entire care continuum of acquired aplastic anemia (AA), from diagnosis and treatment through long-term follow-up, integrating routinely collected clinical data with biospecimens obtained in accordance with applicable ethical and regulatory requirements. The cohort will support observational studies of the natural history of AA; treatment effectiveness and safety; the evolution of clonal hematopoiesis and clonal T-cell populations; immune reconstitution following immunosuppressive therapy (IST); the incidence of platelet transfusion refractoriness and its association with response to IST; and hematopoietic and immune reconstitution after transplantation. Ultimately, the cohort aims to generate real-world evidence to inform clinical practice and reduce the burden of AA.
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