Status:
ACTIVE_NOT_RECRUITING
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
Lead Sponsor:
Vanda Pharmaceuticals
Conditions:
Charcot Marie Tooth Disease (CMT)
Neuromuscular Diseases (NMD)
Eligibility:
FEMALE
Phase:
PHASE1
PHASE2
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 in...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
May 13 2025
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2026
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT07223632
Start Date
May 13 2025
End Date
March 1 2026
Last Update
November 3 2025
Active Locations (1)
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1
Vanda Investigational Site
Madison, Wisconsin, United States, 53792