Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
NCT ID: NCT07223632
Last Updated: 2025-11-03
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
1 participants
INTERVENTIONAL
2025-05-13
2026-03-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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VCA-894A
VCA-894A
intrathecal antisense oligonucleotide injection
Interventions
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VCA-894A
intrathecal antisense oligonucleotide injection
Eligibility Criteria
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Inclusion Criteria
* Genetically confirmed diagnosis of CMT2S with confirmed IGHMBP2 intronic cryptic splice variant c. 1235+894C\>A.
Exclusion Criteria
* Non-reversible conditions that are contraindications to lumbar puncture.
* Pregnancy, recent pregnancy (within 6 weeks), or women who are breastfeeding.
FEMALE
No
Sponsors
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Vanda Pharmaceuticals
INDUSTRY
Responsible Party
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Locations
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Vanda Investigational Site
Madison, Wisconsin, United States
Countries
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References
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Smieszek S, Przychodzen B, Tyner C, Johnson C, Bai H, Kwon JM, Hagan DW, Niccum C, Brighton R, Hawkins K, Aiken R, Nawaz A, Guo X, Hickman J, Polymeropoulos CM, Birznieks G, Polymeropoulos MH. Potential ASO-based personalized treatment for Charcot-Marie-Tooth disease type 2S. Mol Ther Nucleic Acids. 2025 Feb 4;36(1):102479. doi: 10.1016/j.omtn.2025.102479. eCollection 2025 Mar 11.
Other Identifiers
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VP-VCA-894A-2101
Identifier Type: -
Identifier Source: org_study_id
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