Anemia means having too few red blood cells or too little hemoglobin to carry oxygen through the body. It affects about 1.6 billion people worldwide, with causes ranging from iron deficiency to chronic kidney disease to inherited blood disorders. Treatment depends on the underlying cause — iron supplements for deficiency, erythropoiesis-stimulating agents for kidney disease, or more targeted therapies for conditions like sickle cell disease.
What's actually going on in research
Trials are testing new forms of erythropoietin that last longer in the body, iron formulations that work faster with fewer side effects, and drugs that stimulate red blood cell production through different pathways. For inherited anemias like sickle cell disease and beta-thalassemia, gene therapies and gene editing are now FDA-approved. Researchers are also studying hypoxia-inducible factor stabilizers that mimic the body's natural response to low oxygen.
Gene therapy
Two gene therapies were FDA-approved in 2023 for sickle cell disease, offering potential functional cures. Similar approaches are being tested for beta-thalassemia and other inherited forms of anemia.
HIF stabilizers
These pills mimic low-oxygen conditions to boost red blood cell production. Several are approved in other countries for anemia of chronic kidney disease, with U.S. trials ongoing.
Intravenous iron
Newer iron infusions work in a single dose and cause fewer allergic reactions. Trials are comparing them to older formulations and to oral iron in people who don't absorb pills well.
What to know before you search
Eligibility typically depends on hemoglobin level, the underlying cause of anemia, whether kidney disease or cancer is present, and prior treatments tried.
What types of trials are currently open
- Treatment trials — Testing new medications that boost red blood cell production or improve iron delivery, often compared to current standard treatments.
- Gene therapy trials — For inherited anemias, testing one-time treatments that modify a person's own blood stem cells to produce healthy red blood cells.
- Dosing studies — Finding the best dose and schedule for new erythropoietin drugs or iron formulations to balance effectiveness and side effects.
- Quality of life studies — Measuring how treatments affect fatigue, exercise capacity, and daily function in people with chronic anemia.
- Observational studies — Following people with anemia to understand how different causes progress and which treatments work best in real-world practice.
Recently added Anemia trials
HY001N for Patients With Autoimmune Hemolytic Anemia After Failure ≥3 Lines of Therapy.
The goal of this clinical trial is to learn if HY001N cell injection safety and tolerability in adult patients with autoimmune hemolytic anemia after failure of 3 or more lines of therapy. It will also learn about the efficacy of HY001N cell injection to treat adult patients with autoimmune hemolytic anemia. The main questions it aims to answer are: Proportion of participants attaining a CR (defined as normalization of hemoglobin not attributed to transfusion effect and the normalization of hemolytic markers) or CRi (defined as normalization of hemoglobin not attributed to transfusion effect without normalization of hemolytic markers) after HY001N infusion? Proportion of participants attaining a PR (defined as hemoglobin ≥ 100 g/L or at least ≥ 20 g/L increase from baseline not attributed to transfusion effect) after HY001N infusion. • What medical problems do participants have when taking HY001N cell injection? Researchers will see if HY001N cell injection works to treat autoimmune hemolytic anemia. Participants will: * Take apheresis, lymphodepletion regimen and HY001N cell injection. * Visit the clinic on schedule.
Share your health data while taking an anemia medication for kidney disease
This is a single-center, prospective real-world observational study aiming to evaluate the efficacy and safety of oral enarodustat in adult non-dialysis chronic kidney disease (ND-CKD) patients with renal anemia. A total of 90 eligible participants will be enrolled and stratified into three groups according to baseline C-reactive protein (CRP) levels: CRP ≤3 mg/L, 3\<CRP ≤10 mg/L, and CRP\>10 mg/L. All subjects receive routine oral enarodustat treatment with individualized dose titration, together with standard supportive care for CKD. Each participant will be followed up every 4 weeks for a total of 24 weeks. The primary objective is to compare the change in hemoglobin from baseline to week 24 across different inflammation subgroups. Secondary objectives include analyzing dynamic changes of iron metabolism indicators and documenting all adverse events during treatment. This study will explore the optimal individualized dosing strategy of enarodustat under different inflammatory and iron status.
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