Clinical trials / NCT05898620
NCT05898620Phase 3Active not recruiting

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

Study sponsor: Neurogene Inc. · NGNE

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2029-12Estimated
Enrollment · participants33Estimated
Source updated2026-06-10Last update listed in the registry record
Version history

Changes to this trial

Last 12 months
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· 1 field changed

Recruitment status changed

− Before

Recruiting

+ After

Active not recruiting

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· 4 fields changed

Main study measure wording changed

Text changed; whether the study measures changed has not been verified. Saved text may be shortened.

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− Before

Measure: Incidence of Treatment Emergent Adverse Events (TEAEs) Measure: Incidence of Serious Adverse Events (SAEs) Measure: Incidence of Adverse Events of Special Interest (AESIs) Measure: Incidence of clinical laboratory abnormalities Measure: Incidence of new physical and neurologic exam abnormalities

+ After

Measure: Efficacy of NGN-401

Planned enrollment increased by 19 participants

− Before

14 participants (planned)

+ After

33 participants (planned)

Primary completion date changed

− Before

Oct 2029

+ After

Dec 2029

Phase changed

− Before

PHASE1, PHASE2

+ After

PHASE3

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