clinical trial · NCT07223632
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
Charcot Marie Tooth Disease (CMT)Neuromuscular Diseases (NMD)VCA-894A
brief 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.
started
May 13, 2025
primary completion
Mar 1, 2026
completion
Mar 1, 2026
last updated
Nov 3, 2025
official title
The Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient With Confirmed IGHMBP2 Intronic Cryptic Splice Variant C. 1235+894C>A That is Amenable to Antisense Oligonucleotide (ASO)-Mediated Correction of IGHMBP2 Splicing
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol