Aplastic Anemia Clinical Trials

17 recruiting

Aplastic Anemia Trials at a Glance

38 actively recruiting trials for aplastic anemia are listed on ClinicalTrialsFinder across 6 cities in 6 countries. The largest study group is Phase 2 with 23 trials, with the heaviest enrollment activity in Tianjin, Bethesda, and Philadelphia. Lead sponsors running aplastic anemia studies include National Heart, Lung, and Blood Institute (NHLBI), Institute of Hematology & Blood Diseases Hospital, China, and Assistance Publique - Hôpitaux de Paris.

Browse aplastic anemia trials by phase

Treatments under study

About Aplastic Anemia Clinical Trials

Looking for clinical trials for Aplastic Anemia? There are currently 17 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 38 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

Severe Aplastic Anemia (SAA)Hypo-Plastic Myelodysplastic Syndrome (MDS)
National Heart, Lung, and Blood Institute (NHLBI)56 enrolled1 locationNCT03520647
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 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

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
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 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
Recruiting
Phase 2

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Dyskeratosis CongenitaFanconi AnemiaMyelodysplastic Syndromes+5 more
Masonic Cancer Center, University of Minnesota48 enrolled1 locationNCT03579875
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 1

A Reduced-Intensity Conditioning Regimen (Cyclophosphamide, Pentostatin, Anti-thymocyte Globulin) Followed by Haploidentical Hematopoietic Stem Cell Transplant for the Treatment of Patients With Refractory or Recurrent Severe Aplastic Anemia

Recurrent Severe Aplastic AnemiaRefractory Severe Aplastic Anemia
City of Hope Medical Center6 enrolled1 locationNCT05757310
Recruiting
Phase 2

A Study of Emapalumab for Pediatric Aplastic Anemia

Aplastic AnemiaCytopeniaHypocellular Marrow
Memorial Sloan Kettering Cancer Center35 enrolled6 locationsNCT06430788
Recruiting
Phase 2

Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors

Cancer SurvivorsMyelodysplastic SyndromesAplastic Anemia+2 more
Children's Hospital of Philadelphia80 enrolled3 locationsNCT05194397
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

A Prediction Model in Pregnant Women With Aplastic Anemia

PregnancyAplastic AnemiaPrediction Model
Peking University People's Hospital300 enrolled1 locationNCT07101770