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Aplastic Anemia clinical trials
9 of 24 indexed Aplastic Anemia trials are currently recruiting.
24 US clinical trials · 9 currently recruiting
Aplastic Anemia is the subject of 24 registered US clinical trials on ClinicalTrials.gov, 9 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 16 in Phase 1-2 (earlier-stage). The most active sponsor is National Heart, Lung, and Blood Institute (NHLBI), running 2 of these trials.
The research picture
Aplastic Anemia has 24 registered US clinical trials, 9 of them open to new participants right now, about 38% of the total.
- 9
- recruiting participants now
- 38%
- of trials open to enrollment
- 1
- in Phase 3–4 (later-stage)
- 2
- top sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Active & Recent Trials
24 total, page 1 of 1
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
M.D. Anderson Cancer Center
NCT01624805
Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors
Children's Hospital of Philadelphia
NCT05194397
Two Step Haplo With Radiation Conditioning
Thomas Jefferson University
NCT05031897
Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
Washington University School of Medicine
NCT06689800
A Study of Emapalumab for Pediatric Aplastic Anemia
Memorial Sloan Kettering Cancer Center
NCT06430788
Umbilical Cord Blood Transplantation From Unrelated Donors
University of Rochester
NCT03016806
Haploidentical HCT for Severe Aplastic Anemia
St. Jude Children's Research Hospital
NCT04558736
Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia
Fred Hutchinson Cancer Center
NCT06752694
Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
NCT06398457
Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita
Boston Children's Hospital
NCT01659606
Geriatric Optimization Plan to Improve Survival in Older Adult Allogeneic Hematopoietic Cell Transplant Candidates, OTIS Study
Ohio State University Comprehensive Cancer Center
NCT05972577
Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant
M.D. Anderson Cancer Center
NCT03622788
Oral Manifestations of Aplastic Anemia
National Institute of Dental and Craniofacial Research (NIDCR)
NCT00006300
Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
NCT00004464
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Masonic Cancer Center, University of Minnesota
NCT02162420
A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT
Phoenix Children's Hospital
NCT03176849
Stem Cell Factor Medication for Aplastic Anemia
National Heart, Lung, and Blood Institute (NHLBI)
NCT00001398
Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
The Cleveland Clinic
NCT01231841
TPO-Mimetic Use in Children for Hematopoietic Failure
Anjali Sharathkumar
NCT04478227
Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders
Massachusetts General Hospital
NCT01758042
Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia
National Center for Research Resources (NCRR)
NCT00004323
Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization
Jennifer Woyach
NCT04439006
Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia
National Heart, Lung, and Blood Institute (NHLBI)
NCT00767650
Unrelated Cord Blood Transplant Plus a Haplo-Identical (Half-Matched), T-Cell Depleted Stem Transplant From a Related Donor for Subjects With High Risk Malignancies
Joanne Kurtzberg, MD
NCT00673114
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 6 |
| Phase 2 | 10 |
| Phase 4 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Aplastic Anemia Trial Landscape
24 indexed Aplastic Anemia studies · 9 recruiting (38%).
Phase mix: 1 late (III/IV) vs 16 early (I/II).
Top sponsor: National Heart, Lung, and Blood Institute (NHLBI) (2 trials), 9 others listed. 24 shown below, recruiting first.
How to read this condition's trial landscape
That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Aplastic Anemia?
PlainTrial tracks 24 US clinical trials for Aplastic Anemia, of which 9 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Aplastic Anemia?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology - how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.