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NOT YET RECRUITING
NCT07447557
PHASE1/PHASE2

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

Sponsor: Elpida Therapeutics SPC

View on ClinicalTrials.gov

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.

Official title: 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)

Key Details

Gender

All

Age Range

3 Years - 20 Years

Study Type

INTERVENTIONAL

Enrollment

8

Start Date

2026-04

Completion Date

2031-12

Last Updated

2026-03-03

Healthy Volunteers

No

Interventions

GENETIC

ELP-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.