Trial Profile

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)

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 18 Jun 2026

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At a glance

10 Jun 2026 Status changed from recruiting to active, no longer recruiting.
12 May 2026 According to a Neurogene media release, on track to complete dosing in the second quarter of 2026 ~90% of participants have been dosed, the company will present updated interim safety and efficacy data on the pediatric and adolescent/adult cohorts from the Phase 1/2 trial, including at least 12 months of follow-up for all 10 participants, in mid-2026.
26 Feb 2026 According to a Neurogene media release, completion of dosing expected in the second quarter of 2026.

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