A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome
← catalyst calendarNCT05898620 · readout ≤ 1,230 d
Sponsored by Neurogene Inc. (industry) · NGNE — their whole pipeline →.
Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Non randomized · Single · Treatment
Enrollment
33estimated
Sites
16
Countries
Australia, United Kingdom, United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2023-06-13 | actual | When the study began enrolling |
| Primary completion | Dec 2029 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Dec 2029 | estimated | The whole study's end, after follow-up |
| First posted | 2023-06-12 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-06-10 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Rett Syndrome
Intervention
- Genetic: NGN-401
Primary outcome
what the primary-completion date above is the date OFEfficacy of NGN-401
measured 52 Weeks
Publications
- PMID 40173263 — linked by the registry
- PMID 38723617 — linked by the registry
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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