Anemia and Haematopoietic stem-cell transplantation (HSCT): what the trial registry contains
The ClinicalTrials.gov registry holds 163 records for HSCT in Anemia: 26 recruiting now, 12 labelled Phase 3 or 4, across 13 countries. Snapshot 2026-08-29.
Why this is studied in Anemia
Under 'anaemia' the registry mostly holds aplastic anaemia and marrow-failure syndromes, where haematopoietic transplantation is not experimental at all — it is standard care with decades of protocol refinement. Mesenchymal cells appear alongside it as a graft-support and graft-versus-host-disease adjunct, which is a different and much less settled question.
What HSCT means in this table
Haematopoietic transplantation is the oldest and most consequential cell therapy in medicine, with conditioning regimens that carry real mortality risk alongside genuine disease-modifying power. Registrations under this label therefore mix decades-old standard-of-care protocols with newer autoimmune-reset trials, and the risk profile is nothing like that of an outpatient infusion.
The numbers
Every figure below comes from one reproducible query (aplastic anemia x hematopoietic stem cell transplantation) run against ClinicalTrials.gov API v2 on 2026-08-29.
| Metric | Value |
|---|---|
| Registered studies (all statuses) | 163 |
| Interventional studies in the sample | 150 |
| Recruiting right now | 26 |
| Completed | 72 |
| Terminated, withdrawn or suspended | 20 |
| Phase 3 or Phase 4 records | 12 |
| Countries with at least one site | 13 |
| Median planned enrolment | 31 participants |
| Largest single planned enrolment | 4,476 participants |
| First / most recent start year | 1993 — 2028 |
How mature is this evidence?
Registration activity here has moved past first-in-human work — 12 records sit at Phase 3 or 4 out of 163, so the open question is whether confirmatory trials report and replicate.
Of those, 72 are marked completed and 20 were terminated, withdrawn or suspended — a stop rate of 12%, worth weighing when a clinic quotes 'ongoing research' as reassurance.
| Phase label | Records | Share |
|---|---|---|
| Phase 2 | 95 | 49% |
| Not applicable | 44 | 23% |
| Phase 1 | 39 | 20% |
| Phase 3 | 10 | 5% |
| Early Phase 1 | 4 | 2% |
| Phase 4 | 2 | 1% |
Median planned enrolment is 31 participants (largest single study: 4,476). Trials of this size can describe safety and generate hypotheses; detecting a modest clinical benefit reliably usually needs substantially larger numbers with a control group.
Where the studies are
The work is distributed across 13 countries; United States alone accounts for about 77% of mentions, so 'internationally researched' can still mean concentrated in a few regulatory environments.
| Country | Studies with a site there |
|---|---|
| United States | 116 |
| China | 13 |
| South Korea | 6 |
| France | 4 |
| Brazil | 3 |
| Canada | 2 |
| Germany | 2 |
| United Kingdom | 2 |
| Norway | 1 |
| Sweden | 1 |
Legal pathway differs by country — see where cell therapy is legal, by country.
Who runs them
Sponsorship leans academic: 32 of the most frequent lead sponsors read as academic or hospital entities and 14 as companies. Who pays shapes what gets measured and what gets published.
- Masonic Cancer Center, University of Minnesota — 12 records
- University of California, San Francisco — 9 records
- Fred Hutchinson Cancer Center — 8 records
- Roswell Park Cancer Institute — 6 records
- Memorial Sloan Kettering Cancer Center — 6 records
- Baylor College of Medicine — 5 records
Representative registered studies
Recruiting studies first, then the most advanced phases. Each identifier links to the registry record, where eligibility criteria, outcome measures and contacts are published.
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT06646497 — Indication of HSCT in Patients With Refractory/Relapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years | Phase 2 | Recruiting | 52 | Assistance Publique - Hôpitaux de Paris | 2025-01-24 |
| NCT06839456 — Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT | Phase 1, Phase 2 | Recruiting | 100 | Children's Hospital of Philadelphia | 2025-03-21 |
| NCT06430788 — A Study of Emapalumab for Pediatric Aplastic Anemia | Phase 2 | Recruiting | 35 | Memorial Sloan Kettering Cancer Center | 2024-05-21 |
| NCT06378060 — Clinical Study on Modified Allogeneic Hematopoietic Stem Cell Transplantation Regimen for Severe Aplastic Anemia | Phase 2 | Recruiting | 30 | Hematology department of the 920th hospital | 2024-03-01 |
| NCT06412497 — MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other | Phase 2 | Recruiting | 60 | Masonic Cancer Center, University of Minnesota | 2024-06-05 |
| NCT05194397 — Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors | Phase 2 | Recruiting | 80 | Children's Hospital of Philadelphia | 2023-02-23 |
| NCT05126849 — Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic | Phase 2 | Recruiting | 31 | Assistance Publique - Hôpitaux de Paris | 2022-01-06 |
| NCT04099966 — AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion | Phase 2 | Recruiting | 20 | Mitchell Cairo | 2021-04-01 |
| NCT05031897 — Two Step Haplo With Radiation Conditioning | Phase 2 | Recruiting | 63 | Thomas Jefferson University | 2021-10-25 |
| NCT04784052 — Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab | Phase 1, Phase 2 | Recruiting | 18 | Porteus, Matthew, MD | 2021-12-07 |
What this page does and does not tell you
Registration counts measure activity, not effect. A study appears here the moment it is registered — before any participant is enrolled, and regardless of whether results are ever posted.
If a clinic offers you this today, the useful question is not "is there research?" but "which of these registered studies is mine part of, and if none, what evidence applies to the exact product I am being sold?" The Provider Question Builder turns that into a scored checklist.
Frequently asked
How many HSCT studies for Anemia are recruiting right now?
26 of 163 registered records were listed as recruiting in the 2026-08-29 snapshot. Recruitment status changes frequently; the registry record is authoritative.
Is HSCT an approved treatment for Anemia?
Registration on ClinicalTrials.gov is not approval. Approval is granted by a regulator for a specific product and indication after review of submitted evidence. 163 registered studies tell you research exists, not that a product cleared review.
How advanced is the research on HSCT for Anemia?
12 of 163 records carry a Phase 3 or Phase 4 label and 72 are marked completed. Registrations run from 1993 to 2028.
Registry and literature counts retrieved 2026-08-29 (ClinicalTrials.gov API v2; PubMed E-utilities). Registration or publication volume measures research activity, not effectiveness or approval. Educational information — not medical advice; verify product, indication, legal pathway and evidence with an independent qualified physician. Page generated 2026-08-29. See our editorial standards.
Download the query data
Query totals are full API matches; status, phase and country counts describe the first up to 400 returned records per query. Query classes overlap and must not be summed as unique trials. Counts are not treatment outcomes. Snapshot: 2026-08-29. Methods and field definitions.