Testing the safety and effectiveness of EN001 for adults with Charcot-Marie-Tooth Disease Type 1A in a two-phase trial Phase 1b open-label dose testing and Phase 2a randomized placebo-controlled study

What this Trial Is About

Researchers are evaluating the safety and effectiveness of EN001 in adults with Charcot-Marie-Tooth Disease type 1A CMT1A, a nerve disorder. This clinical trial includes two stages Phase 1b uses a dose-escalation design to find the maximum tolerated dose and explore safety and efficacy, while Phase 2a is randomized and placebo-controlled to compare EN001s effects with placebo. The study aims to better understand treatment options for people living with CMT1A. In Phase 1b, participants receive EN001 intravenously twice at 4-week intervals with increasing doses to determine the highest safe dose. Phase 2a randomly assigns participants to one of three groups low-dose EN001, high-dose EN001, or placebo, also administered twice over four weeks. After dosing, participants are monitored long-term for safety and treatment effects for up to five years. Participants will undergo regular assessments including symptom scoring CMT Neuropathy Score version 2, nerve conduction tests, physical examinations, laboratory tests, electrocardiograms, and imaging at scheduled visits up to 24 weeks. Safety and adverse reactions are closely monitored, and ongoing follow-up extends for years to evaluate long-term outcomes. The study collects detailed data to assess the impact of EN001 on CMT1A symptoms and patient health.

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)