NCT07447557

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)

• Status: Not Yet Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Elpida Therapeutics SPC · Industry
• Sex: All
• Age: 3 Years – 20 Years
• Healthy volunteers: Not accepted

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.

Detailed description

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. This clinical study is a first-in-human study designed to assess safety and tolerability of ELP-02 in individuals with CMT4J.

Conditions

• Charcot-Marie-Tooth Disease Type 4J

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2026-04-01
• Primary completion: 2031-12-01
• Completion: 2031-12-01
• First posted: 2026-03-03
• Last updated: 2026-03-03

Regulatory

• FDA-regulated drug study