Severe Aplastic Anemia Clinical Trials

15 recruiting

Severe Aplastic Anemia Trials at a Glance

17 actively recruiting trials for severe aplastic anemia are listed on ClinicalTrialsFinder across 6 cities in 2 countries. The largest study group is Phase 2 with 14 trials, with the heaviest enrollment activity in Bethesda, Tianjin, and Minneapolis. Lead sponsors running severe aplastic anemia studies include National Heart, Lung, and Blood Institute (NHLBI), Institute of Hematology & Blood Diseases Hospital, China, and Masonic Cancer Center, University of Minnesota.

Browse severe aplastic anemia trials by phase

Treatments under study

About Severe Aplastic Anemia Clinical Trials

Looking for clinical trials for Severe Aplastic Anemia? There are currently 15 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 Severe 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 Severe 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 117 of 17 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 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 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

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
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

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

Acute LeukemiaSevere Aplastic Anemia
Mitchell Cairo20 enrolled1 locationNCT04099966
Recruiting
Phase 1

CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia

RelapseRefractorySevere Aplastic Anemia
Institute of Hematology & Blood Diseases Hospital, China15 enrolled1 locationNCT06622694
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
Masonic Cancer Center, University of Minnesota60 enrolled1 locationNCT06412497
Recruiting
Phase 2

TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children

Severe Aplastic AnemiaHemoglobinopathy (Disorder)Bone Marrow Failure Syndrome
Johns Hopkins All Children's Hospital17 enrolled1 locationNCT04356469
Recruiting
Phase 2

Reduced Dose of Cyclophosphamide Combined With Standard Immunosuppressive Therapy to Treat Severe Aplastic Anemia

Severe Aplastic AnemiaCyclophosphamide Reduced-dose
Institute of Hematology & Blood Diseases Hospital, China75 enrolled1 locationNCT06695741
Recruiting
Phase 2

Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia

Severe Aplastic Anemia
Hematology department of the 920th hospital30 enrolled1 locationNCT06378060
Recruiting
Phase 2

Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

Severe Aplastic AnemiaPlatelet Transfusion Refractoriness
Institute of Hematology & Blood Diseases Hospital, China20 enrolled1 locationNCT06254560