RecruitingPhase 1Phase 2NCT07447557

Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)

A Phase 1/2 Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of a Single Dose of ELP-02 Delivered Via Lumbar Intrathecal Administration in Charcot-Marie-Tooth-4J (CMT4J)

Sponsor

Elpida Therapeutics SPC

Enrollment

8 participants

Start Date

Jul 1, 2026

Study Type

INTERVENTIONAL

Conditions

Charcot-Marie-Tooth Disease Type 4J

Summary

First-in-human Phase 1/2, open-label study to evaluate the safety, tolerability, and efficacy of a single lumbar intrathecal dose of ELP-02 to individuals with CMT4J.

Eligibility

Min Age: 3 YearsMax Age: 20 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying ELP-02 for people with charcot-marie-tooth disease type 4j. The study is currently recruiting participants at 2 locations. People eligible for this study include aged 3 Years to 20 Years.

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

GENETICELP-02

ELP-02 is an AAV9-based gene therapy vector that expresses the fully functional form of FIG4 under the control of a synthetic promoter. ELP-02 will be delivered intrathecally and is designed to achieve stable, potentially life-long expression of FIG4 in non-dividing cells.

Locations(2)

Stanford University

Palo Alto, California, United States

University of Iowa

Iowa City, Iowa, United States

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT07447557