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Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
Sponsor: Vanda Pharmaceuticals
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.
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
Key Details
Gender
FEMALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
1
Start Date
2025-05-13
Completion Date
2026-03
Last Updated
2025-11-03
Healthy Volunteers
No
Interventions
VCA-894A
intrathecal antisense oligonucleotide injection
Locations (1)
Vanda Investigational Site
Madison, Wisconsin, United States