Condition

Aplastic Anemia clinical trials and research

• 18Clinical trials
• 8NIH research projects
• 4Countries

Newest Aplastic Anemia clinical trials

All recruiting trials (18)

• Isatuximab in adults with refractory severe aplastic anemia

  This study tests whether the drug isatuximab can help adults with refractory severe aplastic anemia who have very low blood counts.

  Recruiting Phase 1/2 Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT07714512

• Brain network changes in adults with anemia measured by fNIRS

  This project will use functional near-infrared spectroscopy (fNIRS) to see if brain network connections are altered in adults aged 18–65 who have anemia.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT07578675

• Daratumumab for refractory aplastic anemia in adults

  This trial tests whether daratumumab can be safely given and help people with refractory aplastic anemia see a benefit.

  Recruiting Phase 1/2 Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT07287228

• High-altitude stem cell and bone marrow transplant registry on the Qinghai-Tibet Plateau

  This project will collect clinical data and blood/bone marrow samples from people having hematopoietic stem cell transplants on the Qinghai‑Tibet Plateau to see how high altitude affects transplant outcomes and recovery.

  Recruiting Affiliated Hospital of Qinghai University · Xining, Qinghai · NCT07205523

• Predicting pregnancy risks for women with aplastic anemia

  This project will try to create a model to predict which pregnant women with aplastic anemia are most likely to have maternal or newborn complications.

  Recruiting Peking University People's Hospital · Beijing · NCT07101770

• Impact of Aplastic Anemia on Reproductive and Mental Health

  This study looks at how aplastic anemia and its treatments affect the reproductive and mental health of young adults aged 18-40.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT06936930

• A tool to improve family communication in pediatric stem cell transplant decisions

  This study is testing a new tool to help families talk better and make decisions together about stem cell transplants for kids aged 8 to 17.

  Recruiting Washington University School of Medicine · St Louis, Missouri · NCT06689800

• Bone-marrow stem cell transplant with post-transplant cyclophosphamide for refractory or relapsed severe aplastic anemia in patients over 40

  This trial tests whether bone-marrow allogeneic stem cell transplants using post-transplant cyclophosphamide can lower graft-versus-host disease and improve outcomes in patients aged 40–60 with refractory or relapsing severe aplastic anemia after first-line immunosuppression.

  Recruiting at 19 sites Phase 2 Assistance Publique - Hôpitaux de Paris · Paris, France and 25 other locations · NCT06646497

• CD7 CAR-T therapy for severe aplastic anemia before stem cell transplant

  This study is testing a new CAR-T cell therapy for people with severe aplastic anemia to see if it can help them before they receive a stem cell transplant.

  Recruiting Early Phase 1 Zhejiang University · Hanzhou, Zhejiang · NCT06633328

• Emapalumab treatment for pediatric aplastic anemia

  This study is testing if a new treatment called emapalumab can help children with severe aplastic anemia do better with their standard treatments.

  Recruiting at 5 sites Phase 2 Memorial Sloan Kettering Cancer Center · New York, New York and 5 other locations · NCT06430788

• Using Darzalex Faspro to lower antibody levels before stem cell transplant

  This study is testing if a new treatment called Darzalex Faspro can help lower harmful antibodies in adults so they can safely receive a stem cell transplant for blood cancers.

  Recruiting Early Phase 1 Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins · Baltimore, Maryland · NCT06398457

• Survey on the use of Revolade tablets in children with aplastic anemia

  This study is testing if Revolade tablets are safe and effective for children aged 6 to 17 with aplastic anemia who haven't received certain treatments yet.

  Recruiting at 17 sites Novartis · Nagoya, Aichi-ken and 16 other locations · NCT06287268

• Exercise and NAD+ Supplementation for Young Cancer Survivors

  This study is testing if combining exercise with a supplement called nicotinamide riboside can help young cancer survivors improve their muscle strength and fitness.

  Recruiting at 3 sites Phase 2 Children's Hospital of Philadelphia · Duarte, California and 2 other locations · NCT05194397

• Reduced-intensity conditioning to lower treatment-related mortality in stem cell transplant patients

  This study is testing a new way to prepare patients for a stem cell transplant to see if it can lower the risk of serious treatment-related complications.

  Recruiting Phase 2 Thomas Jefferson University · Philadephia, Pennsylvania · NCT05031897

• Long-term study of blood diseases in patients in China

  This study is trying to gather detailed information from patients in China with blood diseases like leukemia and multiple myeloma to better understand how these conditions affect their lives and treatment options.

  Recruiting Institute of Hematology & Blood Diseases Hospital, China · Tianjin, Tianjin Municipality · NCT04645199

• Haploidentical transplant for severe aplastic anemia

  This study is testing a new type of stem cell transplant for people with severe aplastic anemia to see if it can help them recover better and reduce the risk of complications.

  Recruiting Phase 2 St. Jude Children's Research Hospital · Memphis, Tennessee · NCT04558736

• Transplanting umbilical cord blood from unrelated donors

  This study is testing different ways to prepare patients for umbilical cord blood stem cell transplants to see how safe and effective the procedure is for them.

  Recruiting University of Rochester · Rochester, New York · NCT03016806

• Treating Aplastic Anemia and Myelodysplastic Syndrome with Combination Therapy

  This study is testing a combination of four treatments to see if it can help people with aplastic anemia or certain types of myelodysplastic syndrome improve their blood counts and overall health.

  Recruiting Phase 2 M.D. Anderson Cancer Center · Houston, Texas · NCT01624805

NIH-funded research (8)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• Emapalumab for children with aplastic anemia

  Doctors will give emapalumab to children with acquired aplastic anemia to try to stop the immune attack on the bone marrow and help it make blood cells.

  NIH research Columbia University Health Sciences · New York, United States · NIH-11457555

• Clinical and immunologic study of pediatric severe aplastic anemia to personalize care

  A pediatric-focused research effort will collect and study immune and clinical data to tailor treatments for children with severe aplastic anemia.

  NIH research Boston Children's Hospital · Boston, United States · NIH-11453341

• Aplastic Anemia Registry to Understand How Immune and Genetic Discoveries Affect Disease

  A collaborative registry study to learn how genetic background and immune factors influence aplastic anemia and related bone marrow failure conditions in patients.

  NIH research Cleveland Clinic Lerner Com-Cwru · Cleveland, United States · NIH-11443431

• Eltombopag: a new way to boost blood stem cells in aplastic anemia

  A new drug approach aims to help people with idiopathic aplastic anemia by stimulating blood-forming stem cells through mechanisms that may not depend on traditional pathways.

  NIH research Cleveland Clinic Lerner Com-Cwru · Cleveland, United States · NIH-11364969

• Understanding how Eltrombopag helps with aplastic anemia

  This research explores how the medication Eltrombopag helps patients with aplastic anemia by looking at its effects on blood-forming cells.

  NIH research Cleveland Clinic Lerner Com-Cwru · Cleveland, United States · NIH-11120856

• Investigating a new treatment for pediatric aplastic anemia

  This study is looking at how well a new treatment called emapalumab works for kids with acquired aplastic anemia, a rare blood disorder where the body stops making enough blood cells, to see if it can help them produce more blood cells and feel better.

  NIH research Sloan-Kettering Inst Can Research · New York, United States · NIH-11105281

• Comparing bone marrow transplantation with immune therapy for severe aplastic anemia in young patients

  This study is looking at whether bone marrow transplants from unrelated donors or immune suppression therapy works better for kids and young adults with newly diagnosed severe aplastic anemia, especially since not everyone has a sibling who can donate bone marrow.

  NIH research Medical College of Wisconsin · Milwaukee, United States · NIH-11060990

• Comparing bone marrow transplantation and immune suppression therapy for severe aplastic anemia in young patients

  This study is looking at whether getting a bone marrow transplant from an unrelated donor or using immune suppression therapy works better for kids and young adults who have just been diagnosed with severe aplastic anemia, especially since not everyone has a matched sibling donor.

  NIH research Boston Children's Hospital · Boston, United States · NIH-11060911

Related topics

Quick answers

How many Aplastic Anemia clinical trials are recruiting right now?

18, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 4 countries. The most sites are in China (8 trials), United States (8 trials), Japan (1 trial), France (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 2: 6, Phase 1/2: 2, Early Phase 1: 2. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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