How strong is the evidence for stem-cell therapy in Primary Immunodeficiency?
ClinicalTrials.gov registrations alone cannot establish how strong the evidence is. Registry activity is relatively mature, with multiple Phase 3/4 registrations: the registry holds 93 records, 35 completed, 7 at Phase 3 or 4, and 17 stopped early (18%). MSC-specific work accounts for 0 of those records. These counts describe research activity and maturity, not benefit, safety or certainty. Snapshot 2026-08-29.
What counts as a real result in Primary Immunodeficiency
Endpoints are immune reconstitution, infection rate and survival — objective and audited. When a clinic cites these successes to justify an unrelated infusion for a different condition, it is using the strongest evidence in the field to sell the weakest.
What we can say from registrations alone
- Volume: 93 registered studies since 1996.
- Maturity: 7 confirmatory-phase records.
- Delivery: 35 completed; 17 terminated, withdrawn or suspended.
- Scale: median planned enrolment 22 participants, largest 2,240.
The completed studies
Completed does not mean published, and published does not mean positive. These are the finished records — the starting point for checking whether results ever appeared.
| 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 |
| 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 |
| 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 |
| NCT02349906 — Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases | Phase 2 | Completed | 106 | medac GmbH | 2015-04 |
| NCT02327351 — TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PID | Phase 2, Phase 3 | Completed | 98 | Federal Research Institute of Pediatric Hematology, Oncology | 2012-07 |
What registrations cannot tell you
Whether results were positive. Whether they were published at all — a large share of registered studies never report outcomes, which biases what the literature appears to show. And whether the product a commercial clinic sells resembles the product studied: cell source, dose, processing and delivery route vary enormously between a registered protocol and an off-protocol injection.
How to use this when someone is selling you a treatment
Ask which specific registered study supports the offer, then compare its intervention description with your quote. If the answer is a general gesture at "Primary Immunodeficiency research", you are being sold the field's activity rather than evidence about the product. The Provider Question Builder turns that gap into questions.
Frequently asked
How strong is the evidence for stem-cell therapy in Primary Immunodeficiency?
ClinicalTrials.gov registrations alone cannot establish how strong the evidence is. Registry activity is relatively mature, with multiple Phase 3/4 registrations: 93 registered records, 7 at Phase 3/4 and 17 stopped early (2026-08-29).
Has any stem-cell product been approved for this condition?
Check the regulator's own register for the exact product name. Marketing frequently blurs approval for one narrow indication into approval in general.
Why do clinics cite hundreds of studies?
Volume is the cheapest form of credibility. Ask instead for the controlled studies of the specific product, and read what outcome they measured.
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.