Trial Profile

A registrational study of NGN-401 Gene Therapy for Treatment of Rett Syndrome

Status: Planning

Phase of Trial: Phase III

Latest Information Update: 24 Nov 2024

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At a glance

Drugs NGN 401 (Primary)
Indications Rett syndrome
Focus Registrational; Therapeutic Use
Sponsors Neurogene Inc

Most Recent Events

11 Nov 2024 According to a Neurogene media release, company anticipate providing an update on the trial design in the first half of 2025.
21 Mar 2024 New trial record
18 Mar 2024 According to a Neurogene media release, as previously planned, amended the protocol, expanding the trial to include more patients in low-dose Cohort 1 and added a high-dose Cohort 2; these updates are expected to provide a robust dataset and inform the design of a future registrational study

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