Trial Profile

A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 05 Jul 2025

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At a glance

05 Jul 2025 Last checked against ClinicalTrials.gov: US National Institutes of Health record.
22 Oct 2024 Status changed from not yet recruiting to recruiting.
19 Sep 2024 Planned initiation date changed from 1 Aug 2024 to 1 Oct 2024.

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