What research stage is stem-cell therapy for Scleroderma (Systemic Sclerosis) at?
Confirmatory-phase work accounts for 5 of the 44 records here; the remainder is dose-finding, safety or observational (2026-08-29).
Phase distribution
| Phase label | Records | Share |
|---|---|---|
| Phase 2 | 20 | 36% |
| Phase 1 | 17 | 31% |
| Not applicable | 10 | 18% |
| Phase 3 | 4 | 7% |
| Early Phase 1 | 3 | 5% |
| Phase 4 | 1 | 2% |
What the phase mix means for Scleroderma (Systemic Sclerosis)
The later-phase footprint here is genuine and instructive: randomised trials compared transplantation against cyclophosphamide with long follow-up, including mortality reporting. Few areas of cell therapy have that quality of comparison.
The later-phase studies themselves
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT00114530 — Scleroderma: Cyclophosphamide or Transplantation | Phase 2, Phase 3 | Completed | 75 | National Institute of Allergy and Infectious Diseases (NIAID | 2005-06 |
| NCT04464434 — Upfront Autologous HSCT Versus Immunosuppression in Early Diffuse Cutaneous Systemic Sclerosis | Phase 4 | Completed | 60 | UMC Utrecht | 2020-09-17 |
| NCT01445821 — Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial | Phase 3 | Terminated | 44 | Northwestern University | 2011-09-15 |
| NCT01347008 — Effect of Sildenafil on the Microcirculatory Blood Flow and Endothelial Progenitor Cells in Systemic Sclerosis | Phase 3 | Completed | 41 | Federal University of São Paulo | 2011-04 |
| NCT03593902 — Cardiac Safe Transplants for Systemic Sclerosis | Phase 2, Phase 3 | Terminated | 9 | Northwestern University | 2018-05-17 |
How to read phase labels
Early phases exist to find harm and set a dose. They are not built to detect benefit, and they routinely produce encouraging-looking numbers that later trials fail to reproduce. Only a confirmatory design with a comparison arm can separate the intervention from everything else happening to a patient.
Median planned enrolment across sampled studies here is 20 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 |
|---|---|---|---|---|---|
| NCT06888973 — Mesenchymal Stem Cells Infusion in Patients With Autoimmune Diseases | Phase 1, Phase 2 | Enrolling By Invitation | 200 | National Institute of Blood and Marrow Transplant (NIBMT), P | 2025-02-01 |
| NCT02516124 — EBMT ADWP Prospective Non Interventional Study : AutoHSCT in SSc Patients | n/a | Completed | 82 | European Society for Blood and Marrow Transplantation | 2012-12 |
| NCT00114530 — Scleroderma: Cyclophosphamide or Transplantation | Phase 2, Phase 3 | Completed | 75 | National Institute of Allergy and Infectious Diseases (NIAID | 2005-06 |
| NCT05960058 — Acceptance and Effectiveness of Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-COV-2) Vaccination in Systemic Sclerosis Patients Treated by Aut | n/a | Unknown | 72 | Assistance Publique - Hôpitaux de Paris | 2023-07 |
| NCT05482594 — Stem Cell Factor, a Potential Biological Marker of Skin Involvement in Systemic Sclerosis? | n/a | Unknown | 65 | Brugmann University Hospital | 2022-01-11 |
Frequently asked
What research stage is stem-cell therapy for Scleroderma (Systemic Sclerosis) at?
5 of 44 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.
More on this condition
- Scleroderma (Systemic Sclerosis): cell-therapy evidence and registered trials
- How much does stem cell therapy for Scleroderma (Systemic Sclerosis) cost? (2026)
- Scleroderma (Systemic Sclerosis) stem cell therapy — your questions answered (2026)
- Scleroderma (Systemic Sclerosis): cell-therapy evidence by therapy class
- MSC vs HSCT for Scleroderma (Systemic Sclerosis): what the registries show