RecruitingPhase 1Phase 2NCT06328712

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)

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)

Sponsor

ENCell

Enrollment

27 participants

Start Date

May 30, 2024

Study Type

INTERVENTIONAL

Conditions

Charcot-Marie-Tooth Disease, Type 1A

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)

Eligibility

Min Age: 19 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called EN001 and a drug called EN001 Placebo for people with charcot-marie-tooth disease, type 1a. The study is currently recruiting participants at 3 locations. People eligible for this study include aged 19 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGEN001

* 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.

DRUGEN001 Placebo

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