Trial Profile

Phase I/IIa Intrathecal Gene Delivery Clinical Trial for IGHMBP2-Related Diseases

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 13 Oct 2025

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At a glance

Drugs ACTX-401 (Primary)
Indications Charcot-Marie-Tooth disease; Respiratory distress syndrome; Spinal muscular atrophy
Focus Adverse reactions

Most Recent Events

30 Sep 2025 Planned End Date changed from 1 Nov 2028 to 1 Jul 2030.
30 Sep 2025 Planned primary completion date changed from 1 Nov 2026 to 1 Jul 2028.
16 Dec 2021 New trial record

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