What research stage is stem-cell therapy for Muscular Dystrophy at?
Confirmatory-phase work accounts for 0 of the 26 records here; the remainder is dose-finding, safety or observational (2026-08-29).
Phase distribution
| Phase label | Records | Share |
|---|---|---|
| Phase 1 | 18 | 53% |
| Phase 2 | 10 | 29% |
| Not applicable | 5 | 15% |
| Early Phase 1 | 1 | 3% |
What the phase mix means for Muscular Dystrophy
Cell-specific registrations remain early. Later-phase activity in these conditions is largely gene-directed rather than cell-replacement, so aggregate 'stem cell' counts overstate the maturity of cell approaches.
The later-phase studies themselves
No registered study for this condition carries a Phase 3 or Phase 4 label in this snapshot. That is the single most useful fact on this page: the confirmatory stage has not been reached.
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 18 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 |
|---|---|---|---|---|---|
| NCT00874783 — Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases | n/a | Recruiting | 120 | Hadassah Medical Organization | 2009-04 |
| NCT02413450 — Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias | n/a | Enrolling By Invitation | 100 | Johns Hopkins University | 2013-08 |
| NCT06328725 — Evaluate the Efficacy and Safety of EN001 in Patients With Duchenne Muscular Dystrophy | Phase 1, Phase 2 | Unknown | 88 | ENCell | 2024-03 |
| NCT06565208 — First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort | Early Phase 1 | Completed | 77 | Satellos Bioscience, Inc. | 2024-08-21 |
| NCT07287189 — Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients | Phase 2 | Recruiting | 51 | Satellos Bioscience, Inc. | 2025-12-08 |
Frequently asked
What research stage is stem-cell therapy for Muscular Dystrophy at?
0 of 26 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.