Aplastic anemia is a rare but serious condition in which the bone marrow fails to produce enough blood cells, leaving patients at risk of severe infections, bleeding, and anemia. Most cases are caused by an autoimmune attack on blood stem cells, though some are inherited or triggered by drugs and toxins.
What's actually going on in research
Allogeneic stem cell transplantation from a matched sibling remains curative for younger patients with a suitable donor, but immunosuppressive therapy with horse antithymocyte globulin and cyclosporine is standard for those without a transplant option. Eltrombopag, a thrombopoietin receptor agonist, is now added to immunosuppression after trials showed it improves response rates. Trials are exploring alternative donors for transplant, novel immunosuppressive combinations, and gene therapy for inherited forms.
Enhanced immunosuppression
Adding eltrombopag to standard horse antithymocyte globulin and cyclosporine is now established as improving response rates, and trials are testing further additions such as eltanexor and other agents.
Alternative donor transplant
For patients lacking a matched sibling donor, trials are refining outcomes with haploidentical (half-matched) family donors and unrelated donors using improved conditioning and graft-versus-host disease prevention.
Gene therapy for inherited forms
Inherited aplastic anemias such as Fanconi anemia are being targeted with gene-correction strategies using CRISPR and viral vectors to restore hematopoietic stem cell function.
What to know before you search
Eligibility depends on disease severity, patient age, donor availability, prior treatment, and whether the cause is inherited or acquired.
What types of trials are currently open
- Immunosuppression trials — Testing new drug combinations to improve response rates in patients not eligible for stem cell transplantation.
- Stem cell transplant trials — Refining donor selection, conditioning regimens, and graft-versus-host disease prevention in transplant-eligible patients.
- Thrombopoietin agonist trials — Studying eltrombopag and related drugs as additions to standard immunosuppression.
- Gene therapy trials — Testing gene-correction approaches for inherited bone marrow failure syndromes.
- Supportive care trials — Evaluating transfusion strategies, infection prophylaxis, and iron chelation in aplastic anemia.
Recently added Aplastic Anemia trials
A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia
Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells. The pathogenesis of AA is complex and involves an abnormal hematopoietic microenvironment, hematopoietic stem cell/progenitor cell deficiencies and immunity disorders. Currently, the standard treatment for AA includes immunosuppressive therapy (IST) based on anti-thymocyte/lymphocyte globulin (ATG/ALG) and cyclosporine A (CsA) or hematopoietic stem cell transplantation (HSCT). Although HLA-identical sibling allogeneic hematopoietic stem cell transplantation is considered the preferred transplant option for patients with severe aplastic anemia (SAA), only less than 30% of patients have an available HLA-matched sibling donor. In recent years, haploidentical hematopoietic cell transplantation (Haplo-HCT) has developed rapidly and has become an important alternative. However, graft failure and graft-versus-host disease (GVHD) remain significant factors limiting its efficacy. Umbilical cord blood (UCB) contains a diverse population of hematopoietic stem cells. Compared with other sources, cord blood-derived hematopoietic stem cells are more primitive, more viable, and possess higher proliferative capacity. Therefore, cord blood transplantation, with its notable clinical therapeutic effects, has become an effective and reliable alternative to peripheral blood or bone marrow transplantation. Currently, some transplant centers worldwide have adopted the coinfusion of UCB units with haplo-HCT (haplo-cord HCT) achieving preliminary efficacy in promoting engraftment and reducing the incidence of GVHD. A retrospective comparative study of haplo-cord HCT versus IST in patients with SAA identified haplo-cord HCT as the sole independent predictor for superior health-related quality of life (HRQoL) (P \< 0.0001). Based on existing research and clinical experience, this study plans to investigate and further evaluate the safety and efficacy of haplo-cord HCT in the treatment of aplastic anemia. Primary endpoints will include overall survival, engraftment rate, disease-free survival, incidence of GVHD, CMV/EBV reactivation rate, donor chimerism dynamics, and immune reconstitution.
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children. The main questions it aims to answer are: * Is APR-2020 safe and well tolerated? * Does APR-2020 modify or correct an underlying genetic condition which causes DBA? * Does APR-2020 reduce or eliminate the need for blood transfusions and/or restore certain blood counts affected by DBA? Participants will: * Take the drug one time as an infusion. * Undergo two rounds of a cellular harvest procedure in which their own cells will be used in the manufacturing of their own participant-specific product. * Initially return to the clinic for two years of follow up at increasingly sparse intervals.
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