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RECRUITING
NCT06328712
PHASE1/PHASE2

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)

Sponsor: ENCell

View on ClinicalTrials.gov

Summary

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)

Official title: 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)

Key Details

Gender

All

Age Range

19 Years - Any

Study Type

INTERVENTIONAL

Enrollment

27

Start Date

2024-05-30

Completion Date

2026-12-31

Last Updated

2026-05-26

Healthy Volunteers

No

Interventions

DRUG

EN001

* Cohort 1: EN001 Low dose administered intravenously (IV) 2 times at 4 week intervals. * Cohort 2: EN001 High dose administered intravenously (IV) 2 times at 4 week intervals.

DRUG

EN001 Placebo

EN001 Placebo administered intravenously (IV) 2 times at 4 week intervals.

Locations (3)

Dongguk University Gyeongju Hospital

Gyeongju, South Korea

Kyung Hee University Hospital at Gangdong

Seoul, South Korea

Samsung Medical Center

Seoul, South Korea