Aplastic Anemia Clinical Trials

43 recruitingLast updated: June 17, 2026

There are 43 actively recruiting aplastic anemia clinical trials across 6 countries. Studies span Phase 2, Phase 1, Early Phase 1, Not Applicable. Top locations include Bethesda, Maryland, United States, Tianjin, Tianjin Municipality, China, Philadelphia, Pennsylvania, United States. Updated daily from ClinicalTrials.gov.


Aplastic Anemia Trials at a Glance

43 actively recruiting trials for aplastic anemia are listed on ClinicalTrialsFinder across 6 cities in 6 countries. The largest study group is Phase 2 with 25 trials, with the heaviest enrollment activity in Bethesda, Tianjin, and Philadelphia. Lead sponsors running aplastic anemia studies include Institute of Hematology & Blood Diseases Hospital, China, National Heart, Lung, and Blood Institute (NHLBI), and Children's Hospital of Philadelphia.

Browse aplastic anemia trials by phase

Treatments under study

About Aplastic Anemia Clinical Trials

Looking for clinical trials for Aplastic Anemia? There are currently 19 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Aplastic Anemia trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Aplastic Anemia clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 43 trials

Recruiting
Phase 2

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Paroxysmal Nocturnal Hemoglobinuria, PNHSevere Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)
National Heart, Lung, and Blood Institute (NHLBI)56 enrolled1 locationNCT03520647
Recruiting

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

Severe Aplastic AnemiaTelomere Biology DisordersInherited Bone Marrow Failure Syndromes
National Heart, Lung, and Blood Institute (NHLBI)1,000 enrolled1 locationNCT05012111
Recruiting

Cancer in Inherited Bone Marrow Failure Syndromes

Diamond-Blackfan AnemiaDyskeratosis CongenitaFanconi Anemia+2 more
National Cancer Institute (NCI)4,000 enrolled2 locationsNCT00027274
Recruiting
Phase 2

MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.

Severe Aplastic AnemiaAcquired Amegakaryocytic ThrombocytopeniaAcquired Pure Red Cell Aplasia+1 more
Masonic Cancer Center, University of Minnesota60 enrolled1 locationNCT06412497
Recruiting

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

Hodgkin LymphomaNon-Hodgkin LymphomaHematologic Malignancies+14 more
Center for International Blood and Marrow Transplant Research99,999 enrolled142 locationsNCT01351545
Recruiting
Not Applicable

Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy

Solid TumorHematologic MalignancySickle Cell Disease+3 more
Washington University School of Medicine60 enrolled1 locationNCT06689800
Recruiting
Phase 2

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

MDS (Myelodysplastic Syndrome)Severe Aplastic Anemia
National Heart, Lung, and Blood Institute (NHLBI)120 enrolled2 locationsNCT01174108
Recruiting
Phase 1Phase 2

JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

Severe Aplastic AnemiaSingle Lineage Cytopenias, T-LGLHypoplastic MDS
National Heart, Lung, and Blood Institute (NHLBI)145 enrolled1 locationNCT05998408
Recruiting

Brain Cognitive Network Abnormalities in Anemia Patients Using fNIRS

Myelodysplastic SyndromesAplastic AnemiaAutoimmune Hemolytic Anemia+1 more
Institute of Hematology & Blood Diseases Hospital, China323 enrolled1 locationNCT07578675
Recruiting
Phase 2

Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years

Aplastic Anemia
Assistance Publique - Hôpitaux de Paris52 enrolled26 locationsNCT06646497
Recruiting
Phase 2

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Severe Aplastic Anemia
Northside Hospital, Inc.20 enrolled2 locationsNCT02828592
Recruiting
Phase 1Phase 2

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

LeukemiaInborn Errors of MetabolismHLH+11 more
Children's Hospital of Philadelphia100 enrolled1 locationNCT06839456
Recruiting
Phase 2

Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA

MDS (Myelodysplastic Syndrome)Aplastic Anemia (AA)
The First Affiliated Hospital of Soochow University66 enrolled1 locationNCT07400341
Recruiting
Phase 2

Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and/or Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome

Myelodysplastic SyndromeAplastic Anemiade Novo Myelodysplastic Syndrome+1 more
M.D. Anderson Cancer Center140 enrolled1 locationNCT01624805
Recruiting
Phase 1Phase 2

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

Myelodysplastic Syndrome (MDS)Severe Aplastic AnemiaHypo-Plastic MDS
National Heart, Lung, and Blood Institute (NHLBI)37 enrolled1 locationNCT03173937
Recruiting
Phase 2

Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

Aplastic Anemia
Fred Hutchinson Cancer Center20 enrolled1 locationNCT06752694
Recruiting

Revolade Tablets Specified Drug-use Survey

Aplastic Anemia
Novartis Pharmaceuticals10 enrolled17 locationsNCT06287268
Recruiting
Phase 2

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

Severe Aplastic Anemia
National Heart, Lung, and Blood Institute (NHLBI)80 enrolled1 locationNCT04304820
Recruiting
Phase 2

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

Severe Aplastic Anemia
Medical College of Wisconsin60 enrolled25 locationsNCT06517641
Recruiting

National Longitudinal Cohort of Hematological Diseases

Acute Myeloid LeukemiaMultiple MyelomaMyelodysplastic Syndrome+14 more
Institute of Hematology & Blood Diseases Hospital, China2,300 enrolled1 locationNCT04645199