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drugset / Trial / NCT07447557

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

NCT07447557 ↗

Phase 1/2 Recruiting 8 enrolled Elpida Therapeutics SPC
NaSingle-groupOpen-labelTreatment

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.

Timeline

Start
2026-07
Primary completion
2032-12
Completion
2032-12

Drugs

EvaluationDrugModalityDoseRoute
Subject ELP-02 Gene therapy (AAV / viral vector) — Intrathecal

Indications

Charcot-Marie-Tooth disease type 4J