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ClinicalTrials.gov 9 recruiting now official registry

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)
Phase 16Phase 210Phase 41

Active & Recent Trials

24 total, page 1 of 1

RECRUITING Phase 2 140 participants

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

RECRUITING Phase 2 80 participants

Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors

Children's Hospital of Philadelphia

NCT05194397

RECRUITING Phase 2 63 participants

Two Step Haplo With Radiation Conditioning

Thomas Jefferson University

NCT05031897

RECRUITING NA 60 participants

Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy

Washington University School of Medicine

NCT06689800

RECRUITING Phase 2 35 participants

A Study of Emapalumab for Pediatric Aplastic Anemia

Memorial Sloan Kettering Cancer Center

NCT06430788

RECRUITING 30 participants

Umbilical Cord Blood Transplantation From Unrelated Donors

University of Rochester

NCT03016806

RECRUITING Phase 2 21 participants

Haploidentical HCT for Severe Aplastic Anemia

St. Jude Children's Research Hospital

NCT04558736

RECRUITING Phase 2 20 participants

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

RECRUITING Early Phase 1 8 participants

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

ACTIVE NOT RECRUITING Phase 2 40 participants

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Boston Children's Hospital

NCT01659606

ACTIVE NOT RECRUITING NA 30 participants

Geriatric Optimization Plan to Improve Survival in Older Adult Allogeneic Hematopoietic Cell Transplant Candidates, OTIS Study

Ohio State University Comprehensive Cancer Center

NCT05972577

ACTIVE NOT RECRUITING Phase 1 16 participants

Cytokine-Treated Veto Cells in Treating Patients With Hematologic Malignancies Following Stem Cell Transplant

M.D. Anderson Cancer Center

NCT03622788

COMPLETED 146 participants

Oral Manifestations of Aplastic Anemia

National Institute of Dental and Craniofacial Research (NIDCR)

NCT00006300

COMPLETED Phase 2 69 participants

Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

NCT00004464

COMPLETED NA 61 participants

Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia

Masonic Cancer Center, University of Minnesota

NCT02162420

COMPLETED Phase 4 49 participants

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

Phoenix Children's Hospital

NCT03176849

COMPLETED Phase 1 40 participants

Stem Cell Factor Medication for Aplastic Anemia

National Heart, Lung, and Blood Institute (NHLBI)

NCT00001398

COMPLETED Phase 2 20 participants

Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia

The Cleveland Clinic

NCT01231841

COMPLETED Early Phase 1 15 participants

TPO-Mimetic Use in Children for Hematopoietic Failure

Anjali Sharathkumar

NCT04478227

COMPLETED NA 10 participants

Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders

Massachusetts General Hospital

NCT01758042

COMPLETED Phase 2 10 participants

Phase II Study of Bone Marrow Transplantation Using Related Donors in Patients With Aplastic Anemia

National Center for Research Resources (NCRR)

NCT00004323

COMPLETED Phase 1 10 participants

Ibrutinib for the Treatment of COVID-19 in Patients Requiring Hospitalization

Jennifer Woyach

NCT04439006

COMPLETED 7 participants

Neuropsychological Effects of Immunosuppressive Treatment in Subjects With Aplastic Anemia

National Heart, Lung, and Blood Institute (NHLBI)

NCT00767650

COMPLETED Phase 1 3 participants

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

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.

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.

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.

Verify with NIH →

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.