Anemia means you don't have enough healthy red blood cells to carry oxygen through your body. It affects about 1.6 billion people worldwide and has many causes — iron deficiency, chronic disease, inherited conditions like sickle cell disease, kidney failure, cancer treatment. Treatment depends entirely on the underlying cause.
What's actually going on in research
Trials are testing drugs that stimulate red blood cell production without needing injections, gene therapies for inherited anemias like sickle cell disease and beta-thalassemia, iron formulations that work faster with fewer side effects, and treatments for anemia caused by cancer and kidney disease. Researchers are also studying how to predict which patients will respond to specific treatments.
Gene therapy for inherited anemias
Gene therapies for sickle cell disease and beta-thalassemia have been approved by the FDA, offering potential functional cures for some patients. Additional gene-editing approaches are in trials to expand access and durability.
HIF pathway drugs
Pills that mimic the body's low-oxygen response are now approved for anemia caused by chronic kidney disease. They work differently than erythropoietin injections and may have advantages in certain patients.
Novel iron formulations
New iron preparations aim to replenish stores faster with fewer gastrointestinal side effects than standard oral iron. Some can be given as a single infusion rather than repeated doses.
What to know before you search
Eligibility depends on the type and severity of anemia, underlying cause, hemoglobin level, transfusion history, and whether standard treatments have worked.
What types of trials are currently open
- Gene therapy trials — Testing one-time treatments that modify or replace faulty genes in sickle cell disease, beta-thalassemia, and other inherited anemias.
- Treatment trials — Testing new drugs to stimulate red blood cell production or improve iron absorption and utilization.
- Supportive care trials — Studies of transfusion strategies, iron replacement timing, and management of anemia in chronic disease or cancer treatment.
- Biomarker studies — Research to identify which patients will respond to specific anemia treatments based on blood markers or genetic factors.
Recently added Anemia trials
Congenital Hemolytic and Dyserythropoietic Anemias
The main reason for this research study is to further understand how some red blood cells are formed incorrectly or they have an abnormal metabolism in a way that they break easier in the circulation or during their passage through the spleen. Participants and/or family members diagnosed with non-immune hemolytic anemia due to a genetic disorder, such as, hemoglobin disorder, erythrocyte membrane skeleton disorders (e.g. spherocytosis, elliptocytosis, or stomatocytosis) or hydration defect (e.g. xerocytosis, overhydrocytosis) or red blood cell (RBC) enzyme disorders, or with a congenital dyserythropoietic anemia (CDA) will be asked to participate.
MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients
This study being done to learn more about the use of medical Magnetic Resonance Imaging (mMRI) and dedicated dental MRI (ddMRI) as a non-invasive diagnosing tool when evaluating potential oral cancerous and precancerous lesions in Fanconi Anemia patients.
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