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

Name: Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient — clinical trial record (NCT07223632)
Creator: DataLookout
License: https://creativecommons.org/licenses/by/4.0/

StatusActive, Not Recruiting

PhasePhase 1/2

SponsorVanda Pharmaceuticals

Started2025-05-13

View on ClinicalTrials.gov ↗

Trial flagged as At Risk

Primary completion moved at least 11 months later Mar 2026 → Mar 2027

See other at-risk trials from Vanda Pharmaceuticals

Amendment history

2026-08-17

Study Status, Outcome Measures v1

Primary completion pushed: 2026-03 → 2027-03

Completion pushed: 2026-03 → 2027-03

Secondary endpointsdetails revised at 1 of 1 entry

2025-10-30

Original filing

Vanda Pharmaceuticals

23 trials monitored

See 3 trials at risk

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	2027-03 (estimated)
Study Completion	2027-03 (estimated)
Updated on ClinicalTrials.gov	2026-08-19