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Severe Aplastic Anemia: early-phase trial registry
11 of 17 indexed Severe Aplastic Anemia trials are currently recruiting, with more weight in Phase 1-2.
Corpus placement: #1,004 of 28,707 conditions by registered trial count.
17 US clinical trials · 11 currently recruiting · #1,004 of 28,707 conditions by registered trial count
Severe Aplastic Anemia: 17 US clinical trials tracked, 11 recruiting.
Severe Aplastic Anemia is the subject of 17 registered US clinical trials on ClinicalTrials.gov, 11 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 14 in Phase 1-2 (earlier-stage). The most active sponsor is National Heart, Lung, and Blood Institute (NHLBI), running 7 of these trials.
Enrollment posture brief
Registry condition severe-aplastic-anemia
OPEN 11 | RECR 65% | LATE 1 | EARLY 14 | RANK #1004 | SPON 7
Volume-matched condition peers
Nearest open-share peer: Autism Spectrum Disorder (ASD) (65% recruiting · 17 trials)
Severe Aplastic Anemia lists 11 open slots (65% of 17 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Severe Aplastic Anemia on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Severe Aplastic Anemia is recruiting above its size-band peers.
11 of 17 indexed trials (64.7%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Severe Aplastic Anemia research.
National Heart, Lung, and Blood Institute (NHLBI) accounts for 7 of 17 trials (41.2%). 10 distinct sponsors appear on at least one study for this condition label.
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Most Severe Aplastic Anemia trials plan to enrol fewer than 100 people.
Median target enrolment is 53 among the 17 trials that report a genuine target (sentinel values excluded). 13 of those (76.5%) plan for fewer than 100 participants.
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Severe Aplastic Anemia research is mostly interventional.
16 interventional and 1 observational studies make up this index (94.1% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and recent trials
17 total, page 1 of 1
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 Francisco
NCT00516152
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 4 |
| Phase 2 | 10 |
| Phase 3 | 1 |
Top Sponsors
Named interventions
Most-linked intervention names on Severe Aplastic Anemia records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Severe Aplastic Anemia trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Severe Aplastic Anemia page
17 registered trials, 11 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 11 Severe Aplastic Anemia trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- National Heart, Lung, and Blood Institute (NHLBI) sponsors the most Severe Aplastic Anemia trials on record, review their full trial history before evaluating a specific study. See National Heart, Lung, and Blood Institute (NHLBI)'s trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Severe Aplastic Anemia condition card totals 17 registered trials; most-linked intervention Rabbit ATG; top US site state Maryland. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.