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

NCT07223632 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2025-11-03

No results posted yet for this study

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.

Conditions

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

Interventions

DRUG

VCA-894A

intrathecal antisense oligonucleotide injection

Sponsors & Collaborators

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
FEMALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-05-13
Primary Completion
2026-03-31
Completion
2026-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07223632 on ClinicalTrials.gov