At a glance
ClinicalIndex Comparison RecordPhase 3Active· 33 target
Drug / intervention
NGN-401genetic
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Baseline-Controlled, Open-Label, Multicenter, Single-Arm, Pivotal Study to Evaluate the Efficacy, Safety, and Tolerability of NGN-401 in Subjects With Rett Syndrome (Embolden)
In Brief
A Phase 3 clinical trial evaluating NGN-401 for Rett Syndrome. Active but no longer recruiting, targeting 33 participants across 16 sites in 3 countries.
Detailed Summary
This study will evaluate the efficacy and safety profiles of the investigational gene therapy, NGN-401, in females with typical Rett syndrome.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsRett Syndrome
CountriesAustralia, United Kingdom, United States
Collaborators--
Timeline
Phase 3Active
2024202520262027202820292030
First PostedJun 2023
Enrollment StartJun 2023
TodayJul 2026
Primary CompletionDec 2029
First PostedJun 12, 2023
Enrollment StartJun 13, 2023
Primary CompletionDec 1, 2029
TodayJul 2, 2026
Enrollment to primary: 6.5 yearsPosted 3.1 years agoPrimary completion in 3.4 years
Interventions
NGN-401genetic
NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.