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Active Not RecruitingPhase 1/2NCT07223632

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

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 in...

Lead sponsor: Vanda Pharmaceuticals•1 U.S. locations•View source on ClinicalTrials.gov

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Sponsors

Lead Sponsor

Vanda Pharmaceuticals

Age

All ages

Sex

FEMALE

Healthy Volunteers

No

Inclusion Criteria

•Ability and acceptance to provide written informed consent.
•Genetically confirmed diagnosis of CMT2S with confirmed IGHMBP2 intronic cryptic splice variant c. 1235+894C\>A.

Exclusion Criteria

•Significant clinical deterioration of the patient's neurologic status, as judged by the Investigator.
•Non-reversible conditions that are contraindications to lumbar puncture.
•Pregnancy, recent pregnancy (within 6 weeks), or women who are breastfeeding.

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: November 3, 2025Terms

Trial snapshot

StatusACTIVE_NOT_RECRUITING

PhasePHASE1/PHASE2

Enrollment1

Start dateMay 2025

Last updatedNov 3, 2025

Condition

Charcot Marie Tooth Disease (CMT)Neuromuscular Diseases (NMD)

Locations shown

Vanda Investigational Site

Madison, Wisconsin

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: November 3, 2025Data synced to Clareo: May 3, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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