Intellectual Disability: cell-therapy evidence by therapy class
Across every stem-cell intervention, ClinicalTrials.gov holds 27 registered records for Intellectual Disability — 2 recruiting, 4 at Phase 3 or 4, in 12 countries. Snapshot 2026-08-29.
The condition, and why cells are proposed
Intellectual disability is an umbrella covering many distinct genetic and acquired causes, which is why aggregate registry counts here are close to meaningless without knowing the underlying diagnosis. Cell approaches address none of the genetic causes directly; the rationale, where stated, is neuroinflammatory or trophic support.
Therapy classes side by side
| Therapy class | Registered | Recruiting | Phase 3/4 | Countries |
|---|---|---|---|---|
| MSC | 0 | 0 | 0 | 0 |
| HSCT | 17 | 2 | 2 | 11 |
| 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 38% of country mentions among sampled studies; 12 countries host at least one site.
| Country | Studies with a site there |
|---|---|
| United States | 18 |
| France | 6 |
| United Kingdom | 5 |
| Germany | 4 |
| Italy | 4 |
| Argentina | 3 |
| Netherlands | 3 |
| India | 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 |
|---|---|---|---|---|---|
| NCT04090268 — Precision Exercise in Children With Malignant Hemopathies | n/a | Unknown | 380 | University of Milano Bicocca | 2017-04-03 |
| NCT04122742 — Diagnosis of RSTS: Identification of the Acetylation Profiles as Epigenetic Markers for Assessing Causality of CREBBP and EP300 Variants. | n/a | Unknown | 154 | University Hospital, Bordeaux | 2019-10-08 |
| NCT02171104 — MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | Phase 2 | Active Not Recruiting | 149 | Masonic Cancer Center, University of Minnesota | 2014-07-10 |
| NCT00176904 — Stem Cell Transplant for Inborn Errors of Metabolism | Phase 2, Phase 3 | Completed | 135 | Masonic Cancer Center, University of Minnesota | 1995-01 |
| NCT01962415 — Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | Phase 2 | Recruiting | 100 | Paul Szabolcs | 2014-02-04 |
| NCT02698579 — Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product | n/a | Active Not Recruiting | 64 | Genetix Biotherapeutics Inc. | 2016-01-22 |
Most recently registered
| Study | Phase | Status | Planned n | Lead sponsor | Start |
|---|---|---|---|---|---|
| NCT07537374 — A Case-Control Observational Study of Peripheral Blood-Derived iPSC Models to Investigate Oligodendrocyte Lineage Development in Children With William | n/a | Not Yet Recruiting | n/a | Qilu Hospital of Shandong University | 2026-04-01 |
| NCT05665166 — Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II | n/a | Active Not Recruiting | n/a | University of Manchester | 2023-06-01 |
| NCT04528355 — Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC | n/a | Recruiting | n/a | Paul Szabolcs | 2020-08-20 |
| NCT04122742 — Diagnosis of RSTS: Identification of the Acetylation Profiles as Epigenetic Markers for Assessing Causality of CREBBP and EP300 Variants. | n/a | Unknown | n/a | University Hospital, Bordeaux | 2019-10-08 |
| NCT03852498 — A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD) | n/a | Completed | n/a | Genetix Biotherapeutics Inc. | 2019-01-24 |
Reading this honestly
None of these numbers say the therapy works. They say how much organised research exists and how far along it is; effect sizes live in published results, not registry entries. For Intellectual Disability 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 Intellectual Disability?
27 registered records mention a stem-cell intervention for this condition (2026-08-29 snapshot), of which 2 were recruiting.
Which therapy class is furthest along for Intellectual Disability?
Ranked by Phase 3/4 records: HSCT (2), 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.