U.S., March 4 -- ClinicalTrials.gov registry received information related to the study (NCT07447557) titled 'Study of Intrathecal ELP-02 for Charcot-Marie-Tooth Disease Type 4J (CMT4J)' on Jan. 20.
Brief 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.
Study Start Date: April, 2026
Study Type: INTERVENTIONAL
Condition:
Charcot-Marie-Tooth Disease Type 4J
Intervention:
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-l...