Trial Profile

A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 01 Jun 2026

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At a glance

26 May 2026 The protocol has been amended to addition in therapeutic endpoint and addition in in treatment arm size with change in study phase.
26 May 2026 Planned number of patients changed from 12 to 27.
26 May 2026 Planned End Date changed from 1 Oct 2025 to 31 Dec 2026.

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