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Voxelotor increased hemoglobin in sickle cell disease before being withdrawn for safety concerns.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
Give your premature infant iron supplements at specific feeding milestones
The goal of this clinical trial is to determine the serum ferritin level and hematocrit level in very preterm infants receiving donor breast milk(DBM) with an early iron supplementation protocol . The main questions it aims to answer are: Does earlier iron supplementation provide increased ferritin levels at 14 and 28 days of life in subjects fed primarily donor breast milk? Iron supplementation will be implemented when the infant achieves 80ml/kg/day of enteral feeds, as contrasted to the current protocol of full feeds (140 to 160ml/kg/day), the latter usually by Day 14 of life. At day of life(DOL) 14 (± 4 days), a blood sample will be obtained for serum ferritin determination. Similarly, a second blood draw would be performed at day of life 28 (± 4 days) or prior to discharge, whichever comes earlier
Receive iron deficiency screening and treatment before or early in pregnancy
This cluster randomized trial compares preconception with early-pregnancy anemia screening and iron supplementation, assessing late pregnancy iron deficiency anemia and maternal and infant outcomes.
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