What research stage is stem-cell therapy for Anemia at?
Of 195 registrations, 14 reached a confirmatory phase label; everything else is earlier-stage or unlabelled (2026-08-29).
Phase distribution
| Phase label | Records | Share |
|---|---|---|
| Phase 2 | 110 | 47% |
| Phase 1 | 54 | 23% |
| Not applicable | 54 | 23% |
| Phase 3 | 11 | 5% |
| Early Phase 1 | 4 | 2% |
| Phase 4 | 3 | 1% |
What the phase mix means for Anemia
The phase mix looks unusual because established transplant protocols are often registered as Phase 2 or as 'not applicable' comparative studies rather than as new-product Phase 3 trials. Maturity here is carried by decades of registry outcome data outside the phase ladder entirely.
The later-phase studies themselves
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT00003816 — Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer | Phase 2, Phase 3 | Completed | 361 | Roswell Park Cancer Institute | 1998-10-19 |
| NCT07737262 — Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia Patients | Phase 3 | Not Yet Recruiting | 142 | Peking University People's Hospital | 2026-08-01 |
| NCT02838992 — ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia | Phase 4 | Unknown | 130 | Jinan Military General Hospital | 2017-02 |
| NCT02875743 — King's Invasive Aspergillosis Study II | Phase 4 | Completed | 120 | King's College Hospital NHS Trust | 2016-12-07 |
| NCT03413306 — Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA | Phase 3 | Unknown | 100 | Federal Research Institute of Pediatric Hematology, Oncology | 2016-12-10 |
| NCT00352976 — TBI Dose De-escalation for Fanconi Anemia | Phase 2, Phase 3 | Completed | 83 | Masonic Cancer Center, University of Minnesota | 2006-05-18 |
How to read phase labels
Phase 1 asks whether a dose is tolerated, usually in a small group without a control arm. Phase 2 looks for a signal and refines dosing. Phase 3 is confirmatory: larger, controlled, powered to test whether the intervention changes an outcome that matters. Not applicable appears for observational studies or where the sponsor assigned no phase, which is common in cell therapy and not a quality mark either way.
Median planned enrolment across sampled studies here is 30 participants. Small single-arm studies can flag safety problems; they cannot separate a treatment effect from natural fluctuation, placebo response or regression to the mean.
The largest studies by planned enrolment
Size is a crude proxy for what a study can detect, but it separates a serious attempt from a case series presented as evidence.
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT06065852 — National Registry of Rare Kidney Diseases | n/a | Recruiting | 35,000 | UK Kidney Association | 2009-11-06 |
| NCT00212407 — New York Blood Center National Cord Blood Program | Early Phase 1 | Terminated | 4,476 | New York Blood Center | 1993-02 |
| NCT07205523 — High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT) | n/a | Recruiting | 1,000 | Yigeng Cao,MD,PhD | 2023-09-01 |
| NCT00964821 — Flu Vaccine in Preventing Influenza Infection in Healthy Volunteers and in Patients Who Have Undergone Stem Cell Transplant | n/a | Completed | 459 | City of Hope Medical Center | 2007-01 |
| NCT00003816 — Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer | Phase 2, Phase 3 | Completed | 361 | Roswell Park Cancer Institute | 1998-10-19 |
Frequently asked
What research stage is stem-cell therapy for Anemia at?
14 of 195 records are labelled Phase 3 or 4 (2026-08-29).
Is a Phase 3 trial proof that it works?
No — it is a design capable of testing that question. Proof depends on the reported result, replication, and whether the outcome measured matters to patients.
Why do so many records have no phase?
Observational studies and some cell-therapy protocols are registered without a phase label. It is a registry field, not a quality rating.
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.