Anemia — a shortage of healthy red blood cells — has many causes including iron deficiency, chronic disease, kidney failure, bone marrow problems, and inherited conditions. Treatment depends entirely on the cause, which means research spans many different approaches.
What's actually going on in research
Hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitors are oral drugs that stimulate red blood cell production similarly to EPO injections, and several are now used or in late trials for anemia of chronic kidney disease. Luspatercept, a drug that promotes late-stage red cell maturation, has moved into standard care for MDS-related and beta-thalassemia-related anemia and is being tested in other settings. Oral iron formulations and IV iron preparations are being compared in trials to establish the best way to replenish stores in different patient populations.
HIF-PH inhibitors
Oral tablets that activate the body's natural red cell production response are being tested as alternatives to erythropoietin injections for anemia in chronic kidney disease.
Luspatercept
This drug promotes red blood cell maturation in the bone marrow and is being tested across multiple anemia types beyond myelodysplastic syndromes, where it first gained approval.
Iron therapy optimization
Trials are comparing oral and intravenous iron formulations in cancer-related, post-surgical, and inflammatory anemia to find the fastest and safest correction strategies.
What to know before you search
Eligibility depends on anemia cause and hemoglobin level, specific underlying condition (CKD, MDS, etc.), and prior treatment with erythropoiesis-stimulating agents or iron.
What types of trials are currently open
- Drug trials — Testing new erythropoiesis-stimulating agents, iron supplements, or bone marrow stimulants for various anemia types.
- Transfusion trials — Comparing transfusion thresholds and strategies to reduce unnecessary blood transfusions.
- Disease-specific trials — Testing anemia treatment within larger trials for cancer, kidney disease, or bone marrow failure.
- Genetic therapy trials — For inherited anemias, evaluating gene therapy and gene editing approaches.
- Observational studies — Tracking anemia prevalence, causes, and treatment patterns in large populations.
Recently added Anemia trials
Efficacy of Oral Sucrosomial Iron Supplementation in Children With Celiac Disease and Iron Deficiency or Anemia
Celiac disease in children is frequently associated with iron deficiency and/or iron deficiency anemia due to intestinal malabsorption and chronic inflammation. Although a gluten-free diet is the standard treatment and can restore iron balance over time, there is currently no clear evidence or consensus on the role and timing of iron supplementation in pediatric patients at diagnosis. Given the potential impact of anemia on growth and neurodevelopment, strategies that enable a faster correction of iron deficiency are clinically relevant. Sucrosomial® iron has shown improved absorption and gastrointestinal tolerability compared to conventional oral iron in adult celiac patients. This study aims to evaluate whether Sucrosomial® iron supplementation, in addition to a gluten-free diet, is more effective and safe than diet alone in achieving a faster normalization of hemoglobin and iron stores in children with newly diagnosed celiac disease. The primary objective of this randomized, double-blind, placebo-controlled, parallel-group study is to assess whether oral supplementation with Sucrosomial® iron, when added to a gluten-free diet (GFD), accelerates the normalization of iron stores and hemoglobin levels compared with GFD alone in school-age children and adolescents newly diagnosed with celiac disease presenting with hypoferritinemia and/or iron deficiency anemia. Target Study Population: Children and adolescents with celiac disease and iron deficiency or anemia due to iron deficiency. Study Duration Total study duration (per patient) will be about 6 months; total treatment duration (per patient) will be 6 months. Number of Patients: 60 planned Two typologies of patients will be included: with hypoferritinemia and with anemia due to iron deficiency. The randomization process will be stratified, so that: * 15 patients with hypoferritinemia receive active treatment and 15 patients receive placebo; * 15 patients with anemia due to iron deficiency receive active treatment and 15 patients receive placebo. The age of patients will also be considered for the randomization (to assign the correct number of product bottles).
Proton-Based Total Marrow Irradiation for Allogeneic Transplantation in High-Risk AML/MDS
This is an open-label, single-center, non-randomized phase I/II pilot study evaluating proton-based Total Marrow Irradiation (TMI) as part of the conditioning regimen prior to allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adult patients with high-risk or relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). These patients have an unfavorable prognosis with standard conditioning approaches. Participants will receive a standard conditioning regimen consisting of either myeloablative or reduced-intensity chemotherapy, selected according to age and comorbidities, combined with proton TMI delivered at a total dose of 12 Gy in three fractions. Graft-versus-host disease (GvHD) prophylaxis will be administered according to institutional standards, preferentially using post-transplant cyclophosphamide. Patients will subsequently undergo standard allo-HSCT and will be followed for at least 24 months after transplantation. The primary objective of the study is to assess the safety and tolerability of proton TMI added to standard conditioning, as measured by non-relapse mortality and treatment-related toxicity within the first 100 days after transplantation. Secondary objectives include evaluation of engraftment kinetics, incidence of relapse, overall and relapse-free survival, GvHD outcomes, and quality of life. Study outcomes will be analyzed descriptively and compared with a matched historical cohort.
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