What research stage is stem-cell therapy for Autism Spectrum Disorder at?
1 of 25 registered records carry a Phase 3 or Phase 4 label. The rest sit at earlier phases or carry no phase label at all. Snapshot 2026-08-29.
Phase distribution
| Phase label | Records | Share |
|---|---|---|
| Phase 1 | 13 | 41% |
| Phase 2 | 11 | 34% |
| Not applicable | 6 | 19% |
| Early Phase 1 | 1 | 3% |
| Phase 3 | 1 | 3% |
What the phase mix means for Autism Spectrum Disorder
Almost everything registered is early-phase or observational. Autism has no accepted biological endpoint, so trials rely on behavioural scales that require blinded raters — a design demand that early-phase studies frequently do not meet.
The later-phase studies themselves
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT01740869 — Autologous Bone Marrow Stem Cells for Children With Autism Spectrum Disorders | Phase 3 | Completed | 30 | Hospital Universitario Dr. Jose E. Gonzalez | 2012-11 |
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 37 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 |
|---|---|---|---|---|---|
| NCT02168868 — Proteomic Biomarker Tests in Blood Samples from Children with Autism Spectrum Disorder (ASD) | n/a | Recruiting | 900 | Benjamin Gesundheit | 2013-05 |
| NCT07311213 — GENES AND AUTISM - Induced Pluripotent Stem Cells | n/a | Not Yet Recruiting | 450 | Institut National de la Santé Et de la Recherche Médicale, F | 2026-08-01 |
| NCT04089579 — hCT-MSC in Children With Autism Spectrum Disorder | Phase 2 | Completed | 137 | Joanne Kurtzberg, MD | 2020-10-12 |
| NCT07622316 — GAIP ASD Research Study | Phase 1 | Recruiting | 100 | Greater Atlanta Integrative Pediatrics | 2026-06-01 |
| NCT03724136 — Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study | n/a | Unknown | 100 | MD Stem Cells | 2018-10-24 |
Frequently asked
What research stage is stem-cell therapy for Autism Spectrum Disorder at?
1 of 25 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
- Autism Spectrum Disorder: cell-therapy evidence and registered trials
- How much does stem cell therapy for Autism Spectrum Disorder cost? (2026)
- Autism Spectrum Disorder stem cell therapy — your questions answered (2026)
- Autism Spectrum Disorder: cell-therapy evidence by therapy class
- MSC vs exosomes for Autism Spectrum Disorder: what the registries show