Aplastic anaemia trials in United States

In United States 36 Also abroad 68

24 of 36 trials · From ClinicalTrials.gov, checked 15 November 2026. Ask the study team whether a trial fits you.

Phase 1/2Taking people
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Where
Bethesda
Age
12 years to 120 years
People
160
Run by
National Cancer Institute (NCI)
See details and how to joinNCT06303193
Phase 2Taking people
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
Where
Baltimore, Bethesda
Age
4 years to 80 years
People
120
Run by
National Heart, Lung, and Blood Institute (NHLBI)
See details and how to joinNCT01174108
Phase 1/2Taking people
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
Where
Atlanta, Cleveland, Columbus, Duarte, Fairfax, Houston, Miami, New York, Palo Alto, Seattle
Age
18 years and over
People
60
Run by
Dren Bio
See details and how to joinNCT07712562
Phase 2Taking people
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Where
Bethesda
Age
4 years to 75 years
People
56
Run by
National Heart, Lung, and Blood Institute (NHLBI)
See details and how to joinNCT03520647
Phase 2Taking people
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Where
Valhalla
Age
1 day to 30 years
People
20
Run by
Mitchell Cairo
See details and how to joinNCT04099966
Phase 2Taking people
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Where
Atlanta
Age
1 year to 75 years
People
20
Run by
Northside Hospital, Inc.
See details and how to joinNCT02828592
Early phase 1Taking people
Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Bone Marrow or Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies
Where
Baltimore
Age
18 years and over
People
8
Run by
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
See details and how to joinNCT06398457
Phase 1/2Taking people
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
Where
Bethesda
Age
4 years to 75 years
People
37
Run by
National Heart, Lung, and Blood Institute (NHLBI)
See details and how to joinNCT03173937
Phase 2Taking people
Haploidentical HCT for Severe Aplastic Anemia
Where
Memphis
Age
Up to 21 years
People
21
Run by
St. Jude Children's Research Hospital
See details and how to joinNCT04558736
Phase 2Taking people
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
Where
St. Petersburg
Age
0 years to 21 years
People
17
Run by
Johns Hopkins All Children's Hospital
See details and how to joinNCT04356469
Phase 2Taking people
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
Where
Houston
Age
18 years and over
People
140
Run by
M.D. Anderson Cancer Center
See details and how to joinNCT01624805
Phase 1/2Not yet taking people in United States
Prenatal Transplantation for Fetuses With Fanconi Anemia
Where
San Francisco, Stanford
People
12
Run by
Agnieszka Czechowicz
See details and how to joinNCT07408583
No phaseTaking people
Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
Where
St Louis
Age
8 years and over
People
60
Run by
Washington University School of Medicine
See details and how to joinNCT06689800
Phase 2Taking people
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.
Where
Minneapolis
Age
0 years to 75 years
People
60
Run by
Masonic Cancer Center, University of Minnesota
See details and how to joinNCT06412497
Phase 1/2Taking people
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
Where
Philadelphia
Age
1 month to 25 years
People
100
Run by
Children's Hospital of Philadelphia
See details and how to joinNCT06839456
Phase 2Taking people
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
Where
Bethesda
Age
3 years to 99 years
People
80
Run by
National Heart, Lung, and Blood Institute (NHLBI)
See details and how to joinNCT04304820
Phase 2Taking people
Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
Where
Atlanta, Baltimore, Birmingham, Boston, Chapel Hill, Charlotte, Columbus, Detroit, Duarte, Durham, Los Angeles, Milwaukee, Nashville, New York, Oklahoma City, Philadelphia, Rochester, Salt Lake City, Seattle, Stanford, St Louis, Tampa, Westwood
Age
3 years to 75 years
People
60
Run by
Medical College of Wisconsin
See details and how to joinNCT06517641
Phase 2Taking people
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Where
Minneapolis
Age
Up to 65 years
People
48
Run by
Masonic Cancer Center, University of Minnesota
See details and how to joinNCT03579875
Phase 1Taking people
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
Where
Duarte
Age
Up to 75 years
People
6
Run by
City of Hope Medical Center
See details and how to joinNCT05757310
Phase 2Taking people
A Study of Emapalumab for Pediatric Aplastic Anemia
Where
Cincinnati, Milwaukee, New York, Philadelphia, Richmond
Age
0 years to 25 years
People
35
Run by
Memorial Sloan Kettering Cancer Center
See details and how to joinNCT06430788
Phase 2Taking people
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Where
Pittsburgh
Age
2 months to 55 years
People
100
Run by
Paul Szabolcs
See details and how to joinNCT01962415
Phase 2Taking people
Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors
Where
Duarte, Memphis, Philadelphia
Age
10 years to 30 years
People
80
Run by
Children's Hospital of Philadelphia
See details and how to joinNCT05194397
Phase 2Taking people
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Where
Baltimore
Age
4 months to 50 years
People
27
Run by
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
See details and how to joinNCT04232085
Phase 2Taking people
Two Step Haplo With Radiation Conditioning
Where
Philadephia
Age
18 years and over
People
63
Run by
Thomas Jefferson University
See details and how to joinNCT05031897