Clinical Trial

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

Active, Not Recruiting Phase 1/2
View on ClinicalTrials.gov →
Record status
This record was last updated November 3, 2025 (before its estimated March 2026 completion). Its status may not reflect the trial's current state.
Summary
This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHMBP2, when administered by intrathecal injection.
Trial Details
NCT Number NCT07223632
Lead Sponsor Vanda Pharmaceuticals
Conditions Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Enrollment 1 participants
Start Date 2025-05-13
Primary Completion 2026-03 (estimated)
Study Completion 2026-03 (estimated)
Updated on ClinicalTrials.gov 2025-11-03