Primary Immunodeficiency: cell-therapy evidence by therapy class
Across every stem-cell intervention, ClinicalTrials.gov holds 93 registered records for Primary Immunodeficiency — 13 recruiting, 7 at Phase 3 or 4, in 16 countries. Snapshot 2026-08-29.
The condition, and why cells are proposed
Primary immunodeficiencies are inherited defects of immune development, and for several of them haematopoietic transplantation and gene-modified cell therapy are established curative treatments. This is the part of the field where 'stem cell therapy' genuinely means a licensed or standard-of-care procedure — which makes borrowed credibility elsewhere all the more misleading.
Therapy classes side by side
| Therapy class | Registered | Recruiting | Phase 3/4 | Countries |
|---|---|---|---|---|
| MSC | 0 | 0 | 0 | 0 |
| HSCT | 70 | 10 | 3 | 15 |
| exosomes | 0 | 0 | 0 | 0 |
| PRP | 0 | 0 | 0 | 0 |
Counts come from separate registry queries per class; a study testing two interventions can appear in more than one row, so rows do not sum to the all-interventions total.
Geography of the research
United States accounts for about 66% of country mentions among sampled studies; 16 countries host at least one site.
| Country | Studies with a site there |
|---|---|
| United States | 65 |
| France | 9 |
| United Kingdom | 7 |
| Italy | 5 |
| Canada | 4 |
| Germany | 2 |
| Russia | 2 |
| China | 2 |
The largest registered studies
Sorted by planned enrolment — size is not quality, but a 300-participant trial can answer questions a 12-participant series cannot.
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT04870333 — PROphylaxis for paTiEnts at Risk of COVID-19 infecTion -V | Phase 2, Phase 3 | Completed | 2,240 | Cambridge University Hospitals NHS Foundation Trust | 2021-02-19 |
| NCT01186913 — Natural History Study of SCID Disorders | n/a | Enrolling By Invitation | 690 | National Institute of Allergy and Infectious Diseases (NIAID | 2010-09-02 |
| NCT02064933 — Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990 | n/a | Completed | 305 | National Institute of Allergy and Infectious Diseases (NIAID | 2014-02-02 |
| NCT02065869 — Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant | Phase 1, Phase 2 | Terminated | 187 | Bellicum Pharmaceuticals | 2014-04 |
| NCT00110045 — Caspofungin Acetate in Treating Aspergillosis in Patients With Hematologic Cancer or in Patients Who Have Undergone a Stem Cell Transplant | Phase 2 | Completed | 171 | European Organisation for Research and Treatment of Cancer - | 2005-02 |
| NCT03188419 — Breadth of Donor Options for People With Inherited Diseases Requiring Allogeneic Hematopoietic Stem Cell Transplant in the Era of Alternative Donor Tr | n/a | Completed | 161 | National Cancer Institute (NCI) | 2017-06-14 |
Most recently registered
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT07660783 — Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases | n/a | Not Yet Recruiting | n/a | Fred Hutchinson Cancer Center | 2026-09 |
| NCT07766304 — Long-term Psychological and Cognitive Evaluation of Children Treated With Allogeneic Hematopoietic Stem Cell Transplantation for Immunodeficiency | n/a | Not Yet Recruiting | n/a | Assistance Publique - Hôpitaux de Paris | 2026-08 |
| NCT07284641 — Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regul | n/a | Recruiting | n/a | Paul Szabolcs | 2026-05-04 |
| NCT04370795 — Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Ra | n/a | Withdrawn | n/a | National Institute of Allergy and Infectious Diseases (NIAID | 2025-11-26 |
| NCT06954441 — V-IMMUNE: A Novel Immunoglobulin Therapy for Immunodeficiency | n/a | Recruiting | n/a | On Pharma Importadora, Exportadora e Distribuidora de Medica | 2025-08-15 |
Reading this honestly
Treat this as a map of research activity. It tells you whether a claim has an evidence programme behind it — not whether the treatment helped anyone. For Primary Immunodeficiency specifically, the practical question is which registered study — if any — matches the product a provider is offering, and what its outcome measures actually are.
Frequently asked
How many stem-cell trials exist for Primary Immunodeficiency?
93 registered records mention a stem-cell intervention for this condition (2026-08-29 snapshot), of which 13 were recruiting.
Which therapy class is furthest along for Primary Immunodeficiency?
Ranked by Phase 3/4 records: HSCT (3), MSC (0), exosomes (0), PRP (0).
Does a recruiting trial mean I can be treated?
No. Trials have eligibility criteria, and joining one is not the same as buying a treatment. Paying a clinic for an unproven intervention outside a registered study gives you neither the protections nor the data collection of a trial.
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.