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ClinicalTrials.gov 9 recruiting now early-phase tilt

Aplastic Anemia: early-phase trial registry

9 of 24 indexed Aplastic Anemia trials are currently recruiting, with more weight in Phase 1-2.

Corpus placement: #686 of 28,707 conditions by registered trial count.

24 US clinical trials · 9 currently recruiting · #686 of 28,707 conditions by registered trial count

Aplastic Anemia: 24 US clinical trials tracked, 9 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.

Enrollment posture brief

Registry condition aplastic-anemia

OPEN 9 | RECR 38% | LATE 1 | EARLY 16 | RANK #686 | SPON 2

Nearest open-share peer: Acute-graft-versus-host Disease (38% recruiting · 8 trials)

Aplastic Anemia (this)38%Behavior37.5%Bleeding29.2%Allergic Rhinitis8.3%Adult Acute Myeloid Leukemia0%
Open-enrollment share among volume-matched conditions near Aplastic Anemia

Aplastic Anemia lists 9 open slots (38% of 24 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 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.

  1. Aplastic Anemia is recruiting below its size-band peers.

    9 of 24 indexed trials (37.5%) are open to enrollment, against a 39.7% average across 762 conditions with 20-99 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.

  2. Sponsorship of Aplastic Anemia trials is spread across many organizations.

    National Heart, Lung, and Blood Institute (NHLBI) accounts for 2 of 24 trials (8.3%). 21 distinct sponsors appear on at least one study for this condition label.

  3. Most Aplastic Anemia trials plan to enrol fewer than 100 people.

    Median target enrolment is 30 among the 24 trials that report a genuine target (sentinel values excluded). 22 of those (91.7%) plan for fewer than 100 participants.

  4. Aplastic Anemia research is mostly interventional.

    21 interventional and 3 observational studies make up this index (87.5% interventional). Study type is sponsor-reported on ClinicalTrials.gov.

Phase 16Phase 210Phase 41

Open and 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

Phase Distribution

PhaseTrial count
Phase 1 6
Phase 2 10
Phase 4 1

Named interventions

Most-linked intervention names on Aplastic Anemia records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.

Fludarabine Drug
5 trials
Cyclophosphamide Drug
5 trials
cyclosporine Drug
2 trials
cyclophosphamide Drug
2 trials
Tacrolimus Drug
2 trials
Anti-Thymocyte Globulin Biological
2 trials
Mesna Drug
2 trials
Stem Cell Transplant Biological
1 trials

US sites by state

Facility locations filed on Aplastic Anemia trials. A trial with sites in several states counts in each.

Ohio 5 trials
Maryland 5 trials
Pennsylvania 4 trials
Texas 3 trials
California 3 trials
Wisconsin 2 trials
Washington 2 trials
Tennessee 2 trials

Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.

What to do with this Aplastic Anemia page

24 registered trials, 9 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.

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 Aplastic Anemia condition card totals 24 registered trials; most-linked intervention Fludarabine; top US site state Ohio. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.