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Severe Aplastic Anemia clinical trials
Every US clinical trial registered for Severe Aplastic Anemia — phase mix, recruiting status, and the sponsors running them, straight from the NIH ClinicalTrials.gov registry.
17 US clinical trials · 11 currently recruiting
The research picture
Severe Aplastic Anemia has 17 registered US clinical trials, 11 of them open to new participants right now — about 65% of the total.
- 11
- recruiting participants now
- 65%
- of trials open to enrollment
- 1
- in Phase 3–4 (later-stage)
- 7
- top sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Counts reflect the public ClinicalTrials.gov registry as last mirrored by PlainTrial. Status and phase are reported by each study's sponsor. This is reference information, not medical advice.
Active & Recent Trials
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
National Heart, Lung, and Blood Institute (NHLBI)
NCT05012111
JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure
National Heart, Lung, and Blood Institute (NHLBI)
NCT05998408
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
National Heart, Lung, and Blood Institute (NHLBI)
NCT01174108
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
National Heart, Lung, and Blood Institute (NHLBI)
NCT04304820
Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
Medical College of Wisconsin
NCT06517641
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.
Masonic Cancer Center, University of Minnesota
NCT06412497
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
Masonic Cancer Center, University of Minnesota
NCT03579875
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
National Heart, Lung, and Blood Institute (NHLBI)
NCT03173937
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Northside Hospital
NCT02828592
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Mitchell Cairo
NCT04099966
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
Johns Hopkins All Children's Hospital
NCT04356469
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
National Heart, Lung, and Blood Institute (NHLBI)
NCT01623167
Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
National Heart, Lung, and Blood Institute (NHLBI)
NCT02979873
A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)
Boston Children's Hospital
NCT05600426
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
Diane George
NCT01966367
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Baylor College of Medicine
NCT05236764
Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT
University of California, San Francis
NCT00516152
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 10 |
| Phase 3 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Severe Aplastic Anemia Trial Landscape
ClinicalTrials.gov lists 17 US studies indexed under Severe Aplastic Anemia, and 11 of those are currently open to recruitment — roughly 65% of the total volume on the registry. 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.
The phase distribution for Severe Aplastic Anemia shows 1 late-stage studies (Phase 3 and Phase 4 combined) alongside 14 earlier-phase entries (Phase 1 through Phase 2). 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.
Top sponsor activity for Severe Aplastic Anemia is led by National Heart, Lung, and Blood Institute (NHLBI) with 7 indexed trials, alongside 9 other organizations in the top contributor list. The list on this page surfaces up to 17 of the most relevant recent and active entries, ordered with recruiting studies first so practical options are visible. 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 Severe Aplastic Anemia?
PlainTrial tracks 17 US clinical trials for Severe Aplastic Anemia, of which 11 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Severe 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.