Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
← catalyst calendarNCT07223632 · readout ≤ 224 d
Sponsored by Vanda Pharmaceuticals (industry) · VNDA — their whole pipeline →.
Phase
Phase 1/2
Status
Active, not recruiting
Study type
Interventional
Design
Na · None · Treatment
Enrollment
1actual
Sites
1
Country
United States
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2025-05-13 | actual | When the study began enrolling |
| Primary completion | Mar 2027 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Mar 2027 | estimated | The whole study's end, after follow-up |
| First posted | 2025-11-03 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-08-19 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Conditions
- Charcot Marie Tooth Disease (CMT)
- Neuromuscular Diseases (NMD)
Intervention
- Drug: VCA-894A
Primary outcomes
what the primary-completion date above is the date OFAssessment of safety of therapy with VCA-894A when administered via intrathecal injection, as measured by the incidence of adverse events.
measured 631 days
Assessment of CMT2S symptoms following chronic administration of intrathecal VCA-894A, as determined by the change in the Revised Upper Limb Module for Spinal Muscular Atrophy (RULM).
measured 631 days
Assessment of CMT2S symptoms following chronic administration of intrathecal VCA-894A, as determined by the change in the Hammersmith Functional Motor Scale - Expanded (HFMSE).
measured 631 days
Publications
- PMID 40060931 — cited as background
Linked on the registry record. A “linked by the registry” entry is NLM's own automated PubMed match, not a claim by the sponsor that the paper reports this study.
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